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Sangamo Therapeutics Regains Rights to Hemophilia A Gene Therapy Following Pfizer Exit

NextEdge Admin

02 Jan 2025

2 min 27 sec

Sangamo Therapeutics, announced it will regain full rights to giroctocogene fitelparvovec, an investigational gene therapy for adults with moderately severe to severe hemophilia A, following Pfizer's decision to terminate their collaboration. Despite positive Phase 3 AFFINE trial results and Pfizer's initial plans for regulatory submissions in early 2025, the company chose not to proceed with the Biologics License Application (BLA) and Marketing Authorisation Application (MAA). Sangamo is now exploring new partnerships to advance the program.

The AFFINE trial (NCT04370054) demonstrated that a single infusion of giroctocogene fitelparvovec significantly reduced annualized bleeding rates (ABR) compared to routine Factor VIII prophylaxis (1.24 vs. 4.73, p=0.0040), meeting both primary and secondary endpoints. The therapy was generally well tolerated. The trial’s success was highlighted at the 2024 ASH Annual Meeting and Exposition.

Pfizer's decision marks a transition period until April 2025, during which the program will return to Sangamo. Trial participants will continue monitoring as planned. Sangamo remains committed to advancing the therapy, leveraging its expertise and existing partnerships with Genentech and Astellas, while also progressing its Fabry gene therapy program towards a potential BLA submission in late 2025.

Sangamo’s broader pipeline includes innovative programs for neurological diseases, such as the anticipated enrollment in the Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in mid-2025 and a Clinical Trial Authorisation submission for its prion disease program in Q4 2025. These advancements reflect Sangamo's dedication to pioneering genomic medicines and transforming patient care for rare and serious diseases.