Trial Outcomes & Findings for A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A (NCT NCT03061201)

Last Updated: 2025-09-30

Results Overview

An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. An SAE was defined as any untoward medical occurrence that, at any dose, met one or more of the criteria as follows: death, life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly/birth defect and other situations per protocol. AEs included both SAEs and all non-SAEs.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years)

Results posted on

2025-09-30

Participant Flow

Participants with severe hemophilia A were enrolled in this study and received single infusion of SB-525/ PF-07055480. Participants were followed up for maximum of 5 years post-infusion.

Participant milestones

Participant milestones
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vector genomes per kilogram (vg/kg) on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Overall Study STARTED	2	2	2	5
Overall Study COMPLETED	1	1	2	4
Overall Study NOT COMPLETED	1	1	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vector genomes per kilogram (vg/kg) on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Overall Study Participants Terminated at Month 36	0	1	0	1
Overall Study Lost to Follow-up	1	0	0	0

Baseline Characteristics

Race is reported.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.	Total n=11 Participants Total of all reporting groups
Age, Continuous	30.50 Years STANDARD_DEVIATION 9.192 • n=5 Participants	35.50 Years STANDARD_DEVIATION 16.263 • n=7 Participants	32.00 Years STANDARD_DEVIATION 1.414 • n=5 Participants	26.80 Years STANDARD_DEVIATION 6.301 • n=4 Participants	30 Years STANDARD_DEVIATION 7.937 • n=21 Participants
Sex: Female, Male Female	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
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	5 Participants n=4 Participants	11 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants	2 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants	9 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	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
Race/Ethnicity, Customized Race · Asian	0 Participants n=5 Participants • Race is reported.	1 Participants n=7 Participants • Race is reported.	0 Participants n=5 Participants • Race is reported.	0 Participants n=4 Participants • Race is reported.	1 Participants n=21 Participants • Race is reported.
Race/Ethnicity, Customized Race · White	2 Participants n=5 Participants • Race is reported.	1 Participants n=7 Participants • Race is reported.	2 Participants n=5 Participants • Race is reported.	4 Participants n=4 Participants • Race is reported.	9 Participants n=21 Participants • Race is reported.
Race/Ethnicity, Customized Race · Other	0 Participants n=5 Participants • Race is reported.	0 Participants n=7 Participants • Race is reported.	0 Participants n=5 Participants • Race is reported.	1 Participants n=4 Participants • Race is reported.	1 Participants n=21 Participants • Race is reported.

PRIMARY outcome

Timeframe: From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Participants With AEs	2 Participants	2 Participants	2 Participants	5 Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Participants With SAEs	0 Participants	2 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: At Year 1 (Week 52)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention. Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. For Cohort 1, both participants resumed prophylaxis regimen prior to Week 52, and were excluded from analysis as pre-specified in the Statistical Analysis Plan (SAP).

FVIII activity levels were assessed using chromogenic and one-stage clotting assay and which were analyzed in the central laboratory. In this outcome measure results by chromogenic assay are reported.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by Chromogenic Assay at Year 1 (Week 52)	—	0.90 Percentage of Normal Standard Deviation 0.000	11.90 Percentage of Normal	42.60 Percentage of Normal Standard Deviation 53.473

PRIMARY outcome

Timeframe: At Year 2 (Week 104)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by Chromogenic Assay at Year 2 (Week 104)	—	19.30 Percentage of Normal	8.25 Percentage of Normal	25.44 Percentage of Normal Standard Deviation 27.532

PRIMARY outcome

Timeframe: At Year 3 (Week 156)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by Chromogenic Assay at Year 3 (Week 156)	—	0.90 Percentage of Normal	4.40 Percentage of Normal	25.46 Percentage of Normal Standard Deviation 36.998

PRIMARY outcome

Timeframe: At Year 4 (Week 208)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by Chromogenic Assay at Year 4 (Week 208)	—	0.90 Percentage of Normal	3.20 Percentage of Normal	26.55 Percentage of Normal Standard Deviation 42.489

PRIMARY outcome

Timeframe: At Year 5 (Week 260)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by Chromogenic Assay at Year 5 (Week 260)	—	0.90 Percentage of Normal	3.30 Percentage of Normal	23.55 Percentage of Normal Standard Deviation 36.270

PRIMARY outcome

Timeframe: At Year 1 (Week 52)

FVIII activity levels were assessed using chromogenic and one-stage clotting assay and which were analyzed in the central laboratory. In this outcome measure results by one-stage clotting assay are reported.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by One-Stage Clotting Assay at Year 1 (Week 52)	—	1.75 Percentage of Normal Standard Deviation 1.202	19.90 Percentage of Normal	66.37 Percentage of Normal Standard Deviation 83.793

PRIMARY outcome

Timeframe: At Year 2 (Week 104)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by One-Stage Clotting Assay at Year 2 (Week 104)	—	19.00 Percentage of Normal	13.95 Percentage of Normal	38.86 Percentage of Normal Standard Deviation 36.738

PRIMARY outcome

Timeframe: At Year 3 (Week 156)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by One-Stage Clotting Assay at Year 3 (Week 156)	—	3.30 Percentage of Normal	5.70 Percentage of Normal	40.52 Percentage of Normal Standard Deviation 50.231

PRIMARY outcome

Timeframe: At Year 4 (Week 208)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by One-Stage Clotting Assay at Year 4 (Week 208)	—	4.80 Percentage of Normal	13.80 Percentage of Normal	38.78 Percentage of Normal Standard Deviation 53.790

PRIMARY outcome

Timeframe: At Year 5 (Week 260)

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=1 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Central FVIII Activity Levels by One-Stage Clotting Assay at Year 5 (Week 260)	—	4.00 Percentage of Normal	6.10 Percentage of Normal	41.00 Percentage of Normal Standard Deviation 56.749

PRIMARY outcome

Timeframe: Year 1 (Week 9 through Week 53)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention. As pre-specified in planned analysis in the statistical analysis plan (SAP) of the study, this outcome measure was to be assessed only in Cohort 4.

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by chromogenic assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 1 included assessments from Week 9 through Week 53). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by Chromogenic Assay at Yearly Interval 1 (Week 9 Through Week 53)	67.89 Percentage of Normal Standard Deviation 46.585	—	—	—

PRIMARY outcome

Timeframe: Year 2 (Week 54 through Week 108)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by chromogenic assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 2 included assessments from Week 54 through Week 108). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by Chromogenic Assay at Yearly Interval 2 (Week 54 Through Week 108)	40.38 Percentage of Normal Standard Deviation 41.498	—	—	—

PRIMARY outcome

Timeframe: Year 3 (Week 109 through Week 160)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by chromogenic assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 3 included assessments from Week 109 through Week 160). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by Chromogenic Assay at Yearly Interval 3 (Week 109 Through Week 160)	27.66 Percentage of Normal Standard Deviation 41.589	—	—	—

PRIMARY outcome

Timeframe: Year 4 (Week 161 through Week 212)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention. As pre-specified in planned analysis in the SAP of the study, this outcome measure was to be assessed only in Cohort 4. Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by chromogenic assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 4 included assessments from Week 161 through Week 212). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by Chromogenic Assay at Yearly Interval 4 (Week 161 Through Week 212)	24.31 Percentage of Normal Standard Deviation 34.640	—	—	—

PRIMARY outcome

Timeframe: Year 5 (Week 213 through Week 264)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by chromogenic assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 5 included assessments from Week 213 through Week 264). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by Chromogenic Assay at Yearly Interval 5 (Week 213 Through Week 264)	24.87 Percentage of Normal Standard Deviation 39.509	—	—	—

PRIMARY outcome

Timeframe: Year 1 (Week 9 through Week 53)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by one-stage clotting assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 1 included assessments from Week 9 through Week 53). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by One-Stage Clotting Assay at Yearly Interval 1 (Week 9 Through Week 53)	107.44 Percentage of Normal Standard Deviation 72.860	—	—	—

PRIMARY outcome

Timeframe: Year 2 (Week 54 through Week 108)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by one-stage clotting assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 2 included assessments from Week 54 through Week 108). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by One-Stage Clotting Assay at Yearly Interval 2 (Week 54 Through Week 108)	58.55 Percentage of Normal Standard Deviation 56.752	—	—	—

PRIMARY outcome

Timeframe: Year 3 (Week 109 through Week 160)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by one-stage clotting assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 3 included assessments from Week 109 through Week 160). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by One-Stage Clotting Assay at Yearly Interval 3 (Week 109 Through Week 160)	43.95 Percentage of Normal Standard Deviation 57.466	—	—	—

PRIMARY outcome

Timeframe: Year 4 (Week 161 through Week 212)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by one-stage clotting assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 4 included assessments from Week 161 through Week 212). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by One-Stage Clotting Assay at Yearly Interval 4 (Week 161 Through Week 212)	39.60 Percentage of Normal Standard Deviation 51.623	—	—	—

PRIMARY outcome

Timeframe: Year 5 (Week 213 through Week 264)

FVIII activity levels were analyzed in the central laboratory using chromogenic and one-stage clotting assay. In this outcome measure FVIII activity levels were assessed by one-stage clotting assay. As pre-specified, for each participant geometric mean of central FVIII activity levels was calculated of all eligible FVIII activity measurements for each yearly interval (for this outcome measure: Yearly Interval 5 included assessments from Week 213 through Week 264). Following this mean and standard deviation as summary statistics across all evaluable participants was calculated and is reported as data for this outcome measure.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=4 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Geometric Mean of Central FVIII Activity Levels for Cohort 4 by One-Stage Clotting Assay at Yearly Interval 5 (Week 213 Through Week 264)	38.62 Percentage of Normal Standard Deviation 51.844	—	—	—

SECONDARY outcome

Timeframe: Pre-screening period: 12 months prior to screening; Post-infusion period: 3 weeks post-infusion up to date of day before start of prophylaxis or date of data cut or conclusion date, whichever was earlier (maximum up to 5 years)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention.

Total ABR included both treated and untreated bleeding episodes. In this outcome measure total ABR for pre-screening and post-infusion are reported. Pre-screening total ABR was based on the total number of reported bleeding episodes during 12 months prior to screening visit as reported on CRF's Hemophilia A History page. Screening was 2 months before baseline. Post- infusion total ABR = number of all bleeding episodes starting 3 weeks after investigational product/ PF-07055480 (IP) infusion up to date of day before start of prophylaxis (or date of data cut or conclusion date)/ observation period in years, where observation period in years = date of day before start of prophylaxis or date of data cut or conclusion date - 3 weeks after date of IP infusion + 1)/365.25. For a participant who did not start prophylaxis the data cut date or conclusion date is used.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Total Annualized Bleeding Rate (ABR) Pre-screening total ABR	3.50 Bleeds per year Standard Deviation 0.707	14.00 Bleeds per year Standard Deviation 16.971	1.50 Bleeds per year Standard Deviation 2.121	8.80 Bleeds per year Standard Deviation 8.319
Total Annualized Bleeding Rate (ABR) Post infusion total ABR -until prophylaxis is resumed	7.93 Bleeds per year Standard Deviation 3.139	3.07 Bleeds per year Standard Deviation 2.898	3.24 Bleeds per year Standard Deviation 4.586	1.53 Bleeds per year Standard Deviation 3.235

SECONDARY outcome

Timeframe: Post-infusion period: 3 weeks post-infusion up to date of day before start of prophylaxis or date of data cut or conclusion date, whichever was earlier (maximum up to 5 years)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention.

Total ABR include both treated and untreated bleeding episodes. In this outcome measure total ABR by severity for post-infusion period is reported. Severity was categorized as mild, moderate and severe. Post- infusion total ABR = number of all bleeding episodes starting 3 weeks after IP infusion up to date of day before start of prophylaxis (or date of data cut or conclusion date)/ observation period in years, where observation period in years = date of day before start of prophylaxis or date of data cut or conclusion date - 3 weeks after date of IP infusion + 1)/365.25. For a participant who did not start prophylaxis the data cut date or conclusion date is used.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Total ABR by Severity Severe	0.00 Bleeds per year Standard Deviation 0.000	0.47 Bleeds per year Standard Deviation 0.658	0.00 Bleeds per year Standard Deviation 0.000	0.00 Bleeds per year Standard Deviation 0.000
Total ABR by Severity Mild	7.93 Bleeds per year Standard Deviation 3.139	0.87 Bleeds per year Standard Deviation 0.081	0.17 Bleeds per year Standard Deviation 0.241	1.22 Bleeds per year Standard Deviation 2.725
Total ABR by Severity Moderate	0.00 Bleeds per year Standard Deviation 0.000	1.73 Bleeds per year Standard Deviation 2.159	3.07 Bleeds per year Standard Deviation 4.345	0.31 Bleeds per year Standard Deviation 0.528

SECONDARY outcome

Timeframe: Pre-infusion period: 30 days before screening up to pre-infusion (approximately up to 3.23 months); post-infusion period: 3 weeks post-infusion up to up to date of data cut or conclusion date (maximum up to 5 years)

Population: Safety population included all participants enrolled in this study who received any portion of study intervention.

AIR was calculated and reported for pre-infusion and post-infusion. AIR for pre-infusion was calculated as, a) Excluding prophylaxis: number of FVIII replacement infusions for reasons other than prophylaxis prior to IP infusion/ (\[date of IP infusion - date of screening\] + 30)\] \*365.25, or b) number of FVIII replacement infusions for any reason prior to IP infusion/ (\[date of IP infusion - date of screening\] + 30)\] \*365.25. Screening was 2 months before baseline and baseline was approximately 1-week prior to IP infusion. AIR for post- infusion was calculated as: number of FVIII replacement infusions started at 3 weeks after IP infusion up to date of data cut or conclusion date/ number of days in the observation period for the participant in years, where observation period in years = date of data cut or conclusion date - 3 weeks after date of IP infusion + 1)/365.25.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Annualized Infusion Rate (AIR) Post-infusion AIR	58.95 Infusions per year Standard Deviation 73.97	32.79 Infusions per year Standard Deviation 25.32	29.03 Infusions per year Standard Deviation 33.27	3.56 Infusions per year Standard Deviation 7.55
Annualized Infusion Rate (AIR) Pre-infusion AIR (Excluding Prophylaxis)	0.00 Infusions per year Standard Deviation 0.00	2.10 Infusions per year Standard Deviation 2.97	0.00 Infusions per year Standard Deviation 0.00	1.62 Infusions per year Standard Deviation 2.26
Annualized Infusion Rate (AIR) Pre-infusion AIR	100.43 Infusions per year Standard Deviation 2.74	81.15 Infusions per year Standard Deviation 108.82	87.93 Infusions per year Standard Deviation 118.73	120.86 Infusions per year Standard Deviation 30.04

SECONDARY outcome

Timeframe: Baseline; Weeks 12, 24, and 52; Months 24, 36, 48, and 60

Population: Safety population. Here, "Number Analyzed" =participants evaluable for specified timepoints. Participants from "Cohort 1: PF-07055480 9\*10\^11 vg/kg" and "Cohort 2: PF-07055480 2\*10\^12 vg/kg" did not attend the study visit or missed the EQ-5D-5L assessment at Months 24 and 60 and participants from "Cohort 3: PF-07055480 1\*10\^13 vg/kg" did not attend the study visit or missed the EQ-5D-5L assessment at Month 24; hence, no participants were analyzed for Cohorts 1, 2 and 3 at the mentioned visits.

EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group. It measures 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) of health on a 5-point scale. Each dimension had 5 levels: 1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, and 5= extreme problems. EQ-5D-5L had 2 components: Index score and visual analogue scale (VAS). Index score was obtained, according to the health state defined by the 5 dimensions scores, from the Crosswalk Index value calculator and table lookup document under the target country population. A health state is defined by the combination of one level from each of the 5 dimensions. For this study, weights under the US population were used to obtain the Index score. Index score ranged between 0-1, where higher score indicates a better health state, and lower score indicate worse health state. Baseline was defined as the latest non-missing value before IP infusion.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Baseline	0.902 Units on a scale Standard Deviation 0.139	0.577 Units on a scale Standard Deviation 0.254	0.922 Units on a scale Standard Deviation 0.110	0.946 Units on a scale Standard Deviation 0.122
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Week 12	0.011 Units on a scale Standard Deviation 0.016	0.244 Units on a scale	0.078 Units on a scale Standard Deviation 0.110	0.016 Units on a scale Standard Deviation 0.035
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Week 24	-0.033 Units on a scale Standard Deviation 0.129	0.317 Units on a scale Standard Deviation 0.103	0.078 Units on a scale Standard Deviation 0.110	-0.036 Units on a scale Standard Deviation 0.080
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Week 52	-0.165 Units on a scale Standard Deviation 0.266	0.294 Units on a scale Standard Deviation 0.071	0.000 Units on a scale	-0.002 Units on a scale Standard Deviation 0.004
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Month 24	—	—	—	0.000 Units on a scale Standard Deviation 0.000
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Month 36	-0.099 Units on a scale Standard Deviation 0.140	0.334 Units on a scale	-0.210 Units on a scale	0.000 Units on a scale Standard Deviation 0.000
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Month 48	-0.013 Units on a scale	0.219 Units on a scale	-0.197 Units on a scale	0.000 Units on a scale Standard Deviation 0.000
Change From Baseline in the EuroQol, 5 Dimensions, 5 Levels (EQ-5D-5L) Index Score at Weeks 12, 24 and 52; Months 24, 36, 48 and 60 Change at Month 60	—	—	-0.087 Units on a scale Standard Deviation 0.047	-0.053 Units on a scale Standard Deviation 0.105

SECONDARY outcome

Timeframe: Baseline; Weeks 12, 24, and 52; Months 24, 36, 48, and 60

EQ-5D-5L is a standardized measure of health status developed by the EuroQol Group. It measures 5 dimensions of health on a 5-point scale including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension is assessed with 5 levels: 1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, and 5= extreme problems. EQ-5D-5L had 2 components: Index score and VAS. EQ-5D-5L VAS: Participants were asked to indicate their current health status on a scale of 0 (worst health) to 100 (best imaginable health), higher scores signified better health status. Baseline was defined as the latest non-missing value before IP infusion.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Baseline	75.0 Units on a scale Standard Deviation 7.07	73.5 Units on a scale Standard Deviation 9.19	80.0 Units on a scale Standard Deviation 14.14	92.6 Units on a scale Standard Deviation 10.67
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Week 12	4.5 Units on a scale Standard Deviation 6.36	10.0 Units on a scale	5.0 Units on a scale Standard Deviation 21.21	-0.4 Units on a scale Standard Deviation 5.32
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Week 24	10.0 Units on a scale Standard Deviation 7.07	15.0 Units on a scale Standard Deviation 0.00	19.5 Units on a scale Standard Deviation 14.85	-3.0 Units on a scale Standard Deviation 7.52
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Week 52	-1.0 Units on a scale Standard Deviation 12.73	16.0 Units on a scale Standard Deviation 1.41	-10.0 Units on a scale	-4.2 Units on a scale Standard Deviation 6.57
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Month 24	—	—	—	-4.0 Units on a scale Standard Deviation 5.66
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Month 36	0.0 Units on a scale Standard Deviation 0.0	8.0 Units on a scale	0.0 Units on a scale	-0.7 Units on a scale Standard Deviation 9.02
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Month 48	0.0 Units on a scale	10.0 Units on a scale	0.0 Units on a scale	-3.0 Units on a scale Standard Deviation 3.00
Change From Baseline in the EQ-5D-5L- VAS Score at Week 12, 24, 52 and Month 24, 36, 48, 60 Change at Month 60	—	—	4.5 Units on a scale Standard Deviation 6.36	-1.3 Units on a scale Standard Deviation 9.07

SECONDARY outcome

Timeframe: Baseline (one week prior to IP infusion) up to 5 years post-infusion

Population: Safety population included all participants enrolled in this study who received any portion of study intervention.

Positive FVIII inhibitor was assessed using central laboratory using Nijmegen method of the Bethesda assay in an individual with no prior history of FVIII inhibitor. Inhibitor assay results \>0.6 Bethesda units (BU) were considered as positive. Positive results at any timepoint were considered, even if subsequent inhibitor assessment was negative.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Number of Participants With Positive FVIII Inhibitor Levels During the Study	1 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: All samples: Baseline, Day 7, Week 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 and every month after Week 52 until Month 60 until 3 consecutive negative samples are obtained on a sample-type basis; additional for plasma at 12 hours post-infusion

Population: Safety population included all participants enrolled in this study who received any portion of study intervention. Here, "Number Analyzed" signifies participants evaluable for the specified rows.

Peak (maximum) AAV2/6 vector DNA concentration (vg/mL) in plasma, saliva, semen, stool and urine were analyzed with quantitative real-time polymerase chain reaction (PCR). Participants must achieve 3 consecutive negative results for analysis for each sample.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Peak Value of AAV2/6 (Adeno-associated Vector 2/6) Deoxyribonucleic Acid (DNA) in Plasma, Saliva, Semen, Stool and Urine Plasma	25680000 Vector genome per milliliter (vg/mL) Standard Deviation 26620000	292600000 Vector genome per milliliter (vg/mL) Standard Deviation 383900000	1787000000 Vector genome per milliliter (vg/mL) Standard Deviation 1390000000	3442000000 Vector genome per milliliter (vg/mL) Standard Deviation 2809000000
Peak Value of AAV2/6 (Adeno-associated Vector 2/6) Deoxyribonucleic Acid (DNA) in Plasma, Saliva, Semen, Stool and Urine Saliva	44550 Vector genome per milliliter (vg/mL) Standard Deviation 21710	159500 Vector genome per milliliter (vg/mL) Standard Deviation 89870	1555000 Vector genome per milliliter (vg/mL) Standard Deviation 417200	6972000 Vector genome per milliliter (vg/mL) Standard Deviation 5158000
Peak Value of AAV2/6 (Adeno-associated Vector 2/6) Deoxyribonucleic Acid (DNA) in Plasma, Saliva, Semen, Stool and Urine Semen	14600 Vector genome per milliliter (vg/mL) Standard Deviation NA Standard deviation could not be calculated as a single participant was analyzed.	36500 Vector genome per milliliter (vg/mL) Standard Deviation NA Standard deviation could not be calculated as a single participant was analyzed.	145500 Vector genome per milliliter (vg/mL) Standard Deviation 4950	99780 Vector genome per milliliter (vg/mL) Standard Deviation 151900
Peak Value of AAV2/6 (Adeno-associated Vector 2/6) Deoxyribonucleic Acid (DNA) in Plasma, Saliva, Semen, Stool and Urine Stool	604 Vector genome per milliliter (vg/mL) Standard Deviation 407.3	—	2490 Vector genome per milliliter (vg/mL) Standard Deviation 2461	2277 Vector genome per milliliter (vg/mL) Standard Deviation 2814
Peak Value of AAV2/6 (Adeno-associated Vector 2/6) Deoxyribonucleic Acid (DNA) in Plasma, Saliva, Semen, Stool and Urine Urine	—	—	—	9697 Vector genome per milliliter (vg/mL) Standard Deviation 6166

SECONDARY outcome

Time to peak (maximum) AAV2/6 vector DNA concentration (vg/mL) in plasma, saliva, urine, stool and semen were analyzed with quantitative real-time PCR. Participants must achieve 3 consecutive negative results for analysis for each sample; where negative suggests values under the limit of detection.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Time to Peak Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Urine	—	—	—	9.7 Days Standard Deviation 3.79
Time to Peak Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Plasma	1.5 Days Standard Deviation 0.71	1.0 Days Standard Deviation 0.00	1.0 Days Standard Deviation 0.00	1.4 Days Standard Deviation 0.55
Time to Peak Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Saliva	11.5 Days Standard Deviation 4.95	7.5 Days Standard Deviation 0.71	12.0 Days Standard Deviation 4.24	11.8 Days Standard Deviation 3.96
Time to Peak Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Semen	8.0 Days	15.0 Days	11.0 Days Standard Deviation 4.24	14.4 Days Standard Deviation 8.99
Time to Peak Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Stool	8.0 Days Standard Deviation 0.00	—	7.5 Days Standard Deviation 0.71	17.0 Days Standard Deviation 10.32

SECONDARY outcome

Time to undetectable (negative) value of AAV2/6 vector DNA concentration (vg/mL) in plasma, saliva, urine, stool and semen were analyzed with quantitative real-time PCR. Time to undetectable is defined as the number of days from IP infusion until the first of 3 consecutive specimens under the limit of detection (negative).

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Time to Undetectable (Negative) Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Plasma	22.0 Days Standard Deviation 8.49	15.0 Days Standard Deviation 0.00	56.5 Days Standard Deviation 0.71	84.7 Days Standard Deviation 47.92
Time to Undetectable (Negative) Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Saliva	28.5 Days Standard Deviation 0.71	29.0 Days Standard Deviation 0.00	56.5 Days Standard Deviation 0.71	60.5 Days Standard Deviation 17.62
Time to Undetectable (Negative) Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Semen	18.0 Days Standard Deviation 14.14	15.0 Days	42.0 Days Standard Deviation 18.38	42.2 Days Standard Deviation 27.51
Time to Undetectable (Negative) Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Stool	15.0 Days	15.0 Days	42.0 Days Standard Deviation 18.38	72.6 Days Standard Deviation 62.10
Time to Undetectable (Negative) Value of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Urine	8.0 Days Standard Deviation 0.00	7.5 Days Standard Deviation 0.71	8.5 Days Standard Deviation 0.71	14.8 Days Standard Deviation 8.17

SECONDARY outcome

Time to last of 3 consecutive negative value of AAV2/6 vector DNA concentration (vg/mL) in plasma, saliva, urine, stool and semen were analyzed with quantitative real-time PCR.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Time to Last of 3 Consecutive Negative Values of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Plasma	71.0 Days Standard Deviation 19.80	57.0 Days Standard Deviation 0.00	113.5 Days Standard Deviation 0.71	166.7 Days Standard Deviation 92.95
Time to Last of 3 Consecutive Negative Values of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Saliva	85.0 Days Standard Deviation 0.00	84.5 Days Standard Deviation 0.71	113.5 Days Standard Deviation 0.71	105.3 Days Standard Deviation 27.60
Time to Last of 3 Consecutive Negative Values of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Semen	55.5 Days Standard Deviation 40.31	57.0 Days	115.5 Days Standard Deviation 43.13	95.4 Days Standard Deviation 31.41
Time to Last of 3 Consecutive Negative Values of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Stool	56.0 Days	84.0 Days	115.5 Days Standard Deviation 43.13	185.6 Days Standard Deviation 164.77
Time to Last of 3 Consecutive Negative Values of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Urine	28.5 Days Standard Deviation 0.71	29.0 Days Standard Deviation 0.00	28.5 Days Standard Deviation 0.71	45.4 Days Standard Deviation 14.99

SECONDARY outcome

Time to last positive value prior to first of 3 consecutive negatives of AAV2/6 vector DNA concentration (vg/mL) in plasma, saliva, urine, stool and semen were analyzed with quantitative real-time PCR, where positive suggests values above the limit of detection.

Outcome measures

Outcome measures
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 Participants Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 Participants Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Time to Last Positive Value Prior to First of 3 Consecutive Negatives of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Plasma	11.5 Days Standard Deviation 4.95	7.5 Days Standard Deviation 0.71	28.5 Days Standard Deviation 0.71	47.3 Days Standard Deviation 31.75
Time to Last Positive Value Prior to First of 3 Consecutive Negatives of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Saliva	15.5 Days Standard Deviation 0.71	15.0 Days Standard Deviation 0.00	28.5 Days Standard Deviation 0.71	35.5 Days Standard Deviation 14.34
Time to Last Positive Value Prior to First of 3 Consecutive Negatives of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Semen	15.0 Days	—	21.5 Days Standard Deviation 9.19	18.6 Days Standard Deviation 9.56
Time to Last Positive Value Prior to First of 3 Consecutive Negatives of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Stool	8.0 Days	—	21.5 Days Standard Deviation 9.19	21.4 Days Standard Deviation 9.66
Time to Last Positive Value Prior to First of 3 Consecutive Negatives of AAV2/6 Vector DNA in Plasma, Saliva, Semen, Stool and Urine Urine	—	—	—	9.7 Days Standard Deviation 3.79

Adverse Events

Cohort 1: PF-07055480 9*10^11 vg/kg

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 2: PF-07055480 2*10^12 vg/kg

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 3: PF-07055480 1*10^13 vg/kg

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 4: PF-07055480 3*10^13 vg/kg

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1: PF-07055480 910^11 vg/kg n=2 participants at risk Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vg/kg on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg n=2 participants at risk Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg n=2 participants at risk Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg n=5 participants at risk Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
General disorders Pyrexia	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	20.0% 1/5 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.
Infections and infestations Cellulitis	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	50.0% 1/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/5 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.
Infections and infestations Perineal cellulitis	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	50.0% 1/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/5 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.
Injury, poisoning and procedural complications Thermal burn	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	50.0% 1/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/5 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.
Vascular disorders Hypotension	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	0.00% 0/2 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.	20.0% 1/5 • From Baseline (1 week prior to infusion) up to 5 years post-infusion (approximately 5 Years) Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as SAE in one participant and as non-SAE in another participant, or one participant may have experienced both a serious and non-SAE event during the study. Safety population included all participants who enrolled in this study who received any portion of study intervention.

Other adverse events

Additional Information

Pfizer Clinical Trials.gov Call Center

Pfizer Inc.

Phone: 18007181021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER

Measure	Cohort 1: PF-07055480 910^11 vg/kg Participants received a single intravenous infusion of PF-07055480 9.0\10\^11 vector genomes per kilogram (vg/kg) on Day 1.	Cohort 2: PF-07055480 210^12 vg/kg Participants received a single intravenous infusion of PF-07055480 2.0\10\^12 vg/kg on Day 1.	Cohort 3: PF-07055480 110^13 vg/kg Participants received a single intravenous infusion of PF-07055480 1.0\10\^13 vg/kg on Day 1.	Cohort 4: PF-07055480 310^13 vg/kg Participants received a single intravenous infusion of PF-07055480 3.0\10\^13 vg/kg on Day 1.
Overall Study Participants Terminated at Month 36	0	1	0	1
Overall Study Lost to Follow-up	1	0	0	0