Trial Outcomes & Findings for Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial (NCT NCT03066258)

NCT ID: NCT03066258

Last Updated: 2023-05-16

Results Overview

Participants with ocular and non-ocular AEs and SAEs through 26 weeks (24 weeks following RGX-314 administration)

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 42 participants
• Primary outcome timeframe: 26 weeks (24 weeks following RGX-314 administration)
• Results posted on: 2023-05-16

Participant Flow

Participant milestones

Measure	Cohort 1 3E9 GC (genome copies)/eye of RGX-314 (E means exponential constant) RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF (Vascular endothelial growth factor) protein	Cohort 2 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Overall Study STARTED	6	6	6	12	12
Overall Study COMPLETED	5	6	6	12	11
Overall Study NOT COMPLETED	1	0	0	0	1

Reasons for withdrawal

Measure	Cohort 1 3E9 GC (genome copies)/eye of RGX-314 (E means exponential constant) RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF (Vascular endothelial growth factor) protein	Cohort 2 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Overall Study Death	1	0	0	0	1

Baseline Characteristics

Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial

Baseline characteristics by cohort

Measure	Cohort 1 n=6 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Total n=42 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	12 Participants n=4 Participants	12 Participants n=21 Participants	42 Participants n=10 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	7 Participants n=4 Participants	6 Participants n=21 Participants	22 Participants n=10 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants	5 Participants n=4 Participants	6 Participants n=21 Participants	20 Participants n=10 Participants
Race/Ethnicity, Customized White	6 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	12 Participants n=4 Participants	11 Participants n=21 Participants	41 Participants n=10 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	1 Participants n=10 Participants
Race/Ethnicity, Customized Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	0 Participants n=21 Participants	2 Participants n=10 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	5 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	11 Participants n=4 Participants	12 Participants n=21 Participants	40 Participants n=10 Participants
Region of Enrollment United States	6 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants	12 Participants n=4 Participants	12 Participants n=21 Participants	42 Participants n=10 Participants

PRIMARY outcome

Timeframe: 26 weeks (24 weeks following RGX-314 administration)

Participants with ocular and non-ocular AEs and SAEs through 26 weeks (24 weeks following RGX-314 administration)

Outcome measures

Measure	Cohort 1 n=6 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))	6 Participants	6 Participants	6 Participants	12 Participants	12 Participants

SECONDARY outcome

Timeframe: 106 weeks (104 weeks following RGX-314 administration)

Participants with ocular and non-ocular AEs and SAEs

Outcome measures

Measure	Cohort 1 n=6 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Safety (Participants With Ocular and Non-ocular AEs and SAEs)	6 Participants	6 Participants	6 Participants	12 Participants	12 Participants

SECONDARY outcome

Timeframe: 106 weeks (104 weeks following RGX-314 administration)

Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.

Outcome measures

Measure	Cohort 1 n=5 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=11 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Change From Baseline in BCVA (Best Corrected Visual Acuity)	-7.6 ETDRS letters Interval -16.0 to 6.0	1.2 ETDRS letters Interval -9.0 to 8.0	14.0 ETDRS letters Interval -3.0 to 32.0	0.9 ETDRS letters Interval -45.0 to 17.0	-3.8 ETDRS letters Interval -37.0 to 8.0

SECONDARY outcome

Timeframe: 106 weeks (104 weeks following RGX-314 administration)

Retinal fluid status of the study eye was evaluated using spectral domain OCT (Optical Coherence Tomography). A decrease in value indicates a decrease in fluid

Outcome measures

Measure	Cohort 1 n=5 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=11 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Change From Baseline in CRT (Central Retinal Thickness)	-88.6 μm Interval -248.0 to 11.0	25.3 μm Interval -10.0 to 110.0	2.0 μm Interval -46.0 to 142.0	-56.7 μm Interval -170.0 to 160.0	-108.2 μm Interval -360.0 to 85.0

SECONDARY outcome

Timeframe: 106 weeks (104 weeks following RGX-314 administration)

The number of supplemental anti-VEGF injections given after RGX-314 was administered. Injections per year which were determined by the number of supplemental injections divided total follow-up in study days which is annualized to a per year rate. Injections were given for signs of worsening disease at a study visit, per the discretion of the investigator.

Outcome measures

Measure	Cohort 1 n=6 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Supplemental Injections (Annualized Rate of Supplemental Injections)	10.3 supplemental injections per year Standard Error 0.86	9.3 supplemental injections per year Standard Error 1.94	2.8 supplemental injections per year Standard Error 1.62	4.4 supplemental injections per year Standard Error 1.25	2.0 supplemental injections per year Standard Error 1.07

SECONDARY outcome

Timeframe: 106 weeks (104 weeks following RGX-314 administration)

Area of Choroidal Neovascularization of the study eye was assessed with color fundus photography. Analysis was performed by the central reading center. An increase in value represents an increase in CNV.

Outcome measures

Measure	Cohort 1 n=4 Participants 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=5 Participants 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=5 Participants 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 Participants 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=11 Participants 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Mean Change From Baseline in Area of CNV (Choroidal Neovascularization)	-0.4 mm^2 Standard Deviation 1.62	0.0 mm^2 Standard Deviation 1.24	-1.2 mm^2 Standard Deviation 1.69	-1.1 mm^2 Standard Deviation 3.58	-0.6 mm^2 Standard Deviation 3.30

Adverse Events

Cohort 1

Serious events: 2 serious events

Other events: 5 other events

Deaths: 1 deaths

Cohort 2

Serious events: 2 serious events

Other events: 6 other events

Deaths: 0 deaths

Cohort 3

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Cohort 4

Serious events: 3 serious events

Other events: 12 other events

Deaths: 0 deaths

Cohort 5

Serious events: 4 serious events

Other events: 12 other events

Deaths: 1 deaths

Serious adverse events

Measure	Cohort 1 n=6 participants at risk 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 participants at risk 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 participants at risk 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 participants at risk 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 participants at risk 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Vascular disorders Hypertension	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Metabolism and nutrition disorders Hypervolaemia	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Musculoskeletal and connective tissue disorders Arthritis	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Nervous system disorders Encephalopathy	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Psychiatric disorders Delirium	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Respiratory, thoracic and mediastinal disorders Respiratory Failure	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Retinal Detachment	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Infections and infestations Endophthalmitis	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Visual Impairment	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Cardiac disorders Atrial Fibrillation	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Gastrointestinal disorders Colitis Ischaemic	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
General disorders Asthenia	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Hepatobiliary disorders Cholelithiasis	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Infections and infestations Diverticulitis	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Infections and infestations Pneumonia	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Infections and infestations Urinary Tract Infection	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Injury, poisoning and procedural complications Deep Vein Thrombosis Postoperative	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Injury, poisoning and procedural complications Femoral Neck Fracture	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population

Other adverse events

Measure	Cohort 1 n=6 participants at risk 3E9 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 2 n=6 participants at risk 1E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 3 n=6 participants at risk 6E10 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 4 n=12 participants at risk 1.6E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein	Cohort 5 n=12 participants at risk 2.5E11 GC/eye of RGX-314 RGX-314: RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
Eye disorders Conjunctival Haemorrhage	33.3% 2/6 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	66.7% 4/6 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	75.0% 9/12 • Number of events 9 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	91.7% 11/12 • Number of events 11 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Eye Irritation	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 4/12 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Intraocular Pressure Increased	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 2/6 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 4/12 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Eye Pain	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Visual Acuity Reduced/Impairment	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Retinal Degeneration	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 4/12 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Photopsia	33.3% 2/6 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Conjunctival Hyperaemia	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 2/6 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Vision Blurred	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Retinal pigmentary changes (depigmentation, pigment epitheliopathy & pigmentation)	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	100.0% 12/12 • Number of events 12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	91.7% 11/12 • Number of events 11 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Retinal Haemorrhage	66.7% 4/6 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Intraocular Inflammation	66.7% 4/6 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	50.0% 3/6 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	25.0% 3/12 • Number of events 3 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Metamorphopsia	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Retinal Thickening	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	33.3% 4/12 • Number of events 4 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Dry Age-Related Macular Degeneration	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Erythema Of Eyelid	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Photophobia	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Posterior Capsule Opacification	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 1/6 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/12 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population
Eye disorders Vitreous Floaters	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	0.00% 0/6 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	16.7% 2/12 • Number of events 2 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population	8.3% 1/12 • Number of events 1 • Safety was the primary focus for the initial 24 weeks after RGX-314 administration (primary study period). Following completion of the primary study period, subjects continued to be assessed until 104 weeks following treatment with RGX-314 (Week 106). At the end of the study, subjects were invited to participate in a long-term follow-up study (NCT03999801). Other AEs reported include: RGX-314-emergent Non-serious Ocular Adverse Events Occurring in \>5% of Subjects by Preferred Term, Sorted by Frequency in Total Group As-treated Population SAE reported include: RGX-314-emergent Serious Adverse Events by System Organ Class and Preferred Term As-treated Population

Additional Information

Sherri Van Everen, PharmD/ Vice President of Clinical Development

Regenxbio Inc.

Phone: 240.552.8181

Email: svaneveren@regenxbio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place