Vertex, CRISPR Therapeutics Report $116M in 2025 Casgevy Sales as Adoption Accelerates

Vertex Pharmaceuticals and CRISPR Therapeutics reported that their gene-editing therapy Casgevy (exagamglogene autotemcel) generated $54 million in fourth-quarter revenue and $116 million for full-year 2025, marking the highest quarterly sales since the drug's launch. The results exceeded Wall Street expectations and drove Vertex shares up 6% Friday morning.

A total of 64 patients received infusions in 2025, including 30 during the fourth quarter. Globally, 147 patients initiated treatment through the first cell collection during the year. Patient initiations nearly tripled compared to 109 in 2024, reflecting growing momentum heading into 2026.

Casgevy is the first FDA-approved gene-editing therapy for sickle cell disease and transfusion-dependent beta thalassemia. The process of getting Casgevy can take nearly 12 months from start to infusion. Starting with a patient's blood stem cells, Vertex uses CRISPR gene editing technology to help those cells produce fetal hemoglobin. That production can not only alleviate pain crises, but essentially cure patients of their disease. Yet, the chemotherapy conditioning required to prepare for those infusions can be debilitating and cause side effects such as infertility, which has turned off some potential patients.

Sales could continue to fluctuate quarter to quarter as the scheduling process for infusion is long and patients may wait to receive the final treatment during a preferred time like the holidays. Casgevy could possibly bring in $227 million in sales throughout 2026, given the number of patients who have started cell collection. Vertex expects that pace to "smooth out" by 2027, and is "very encouraged by the robust flow of patients," the Chief Operating Officer said on an earnings call Thursday afternoon.

Access to Casgevy continued to expand across key markets. Reimbursement and access for Casgevy now extend to approximately 90% of eligible patients in the U.S., with rollouts reaching Europe and the Middle East. In January, Vertex secured reimbursed access for sickle cell disease patients in Scotland.

The company received a priority voucher from the Food and Drug Administration to speed up review of Casgevy in patients aged 5 to 11, and said at a medical meeting in December it planned to ask for approval in the first half of 2026. Clearance would likely boost revenue from the gene editing treatment in the coming years.

Vertex notched $12 billion in total revenue for 2025, with about $3.2 billion coming in during the fourth quarter. Overall, sales were up 9% year-over-year. Vertex's revenues are dominated by its arsenal of cystic fibrosis medications, with a little over $10 billion of the $12 billion made in 2025 coming from Trikafta and Kaftrio. The company forecasts roughly $13 billion to $13.1 billion in revenue for this year, including $500 million or more from non-CF products.

Beyond hematology, CRISPR Therapeutics continues to advance its in vivo liver editing programs. CTX310 remains in Phase 1b trials targeting lipid disorders, while CTX321, a next-generation Lp(a) program, is progressing through enabling studies with updates expected in the second half of 2026. Its siRNA-based candidate CTX611, developed with Sirius Therapeutics, is currently in Phase 2 trials for patients undergoing knee replacement surgery and may have broader applications across thromboembolic diseases.

Zugo-cel is also progressing in autoimmune and oncology indications, including systemic lupus erythematosus and B-cell malignancies. The therapy is being evaluated in combination with pirtobrutinib under an existing collaboration.

CRISPR Therapeutics ended 2025 with $1.98 billion in cash and marketable securities. R&D expenses rose to $83.5 million in the fourth quarter, while net loss widened to $130.6 million in the fourth quarter from $37.3 million a year earlier. The company is still loss making, with net income of about $581.6 million loss, and does not yet have meaningful revenue beyond Casgevy. CRISPR Therapeutics shares were up 8.81% at $53.24 at the time of publication on Friday.