Pfizer hands hemophilia A gene therapy program back to Sangamo

Pfizer will terminate its global collaboration and license agreement with Sangamo Therapeutics, handing the development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia, back to the California-based biotech.

The decision seemingly came as a surprise to Sangamo, who, following positive phase 3 trial results announced in July 2024, believed the therapy was “well positioned for regulatory submissions and potential commercialization.” According to Sangamo, in November 2024, Pfizer had indicated that it was discussing the data with regulatory authorities, anticipating BLA and MAA submissions in early 2025.

The two had inked the partnership back in 2017, with Pfizer handing Sangamo $70 million upfront plus the potential for milestone payments of up to $475 million. Now, Sangamo says it intends to explore all options to advance the program, including seeking a potential new collaboration partner.

Giroctocogene fitelparvovec is a novel gene therapy that contains a bio-engineered AAV6 capsid and a modified B-domain deleted human coagulation FVIII gene. The goal of this investigational treatment for people living with hemophilia A is that a single infusion of giroctocogene fitelparvovec may allow them to produce FVIII themselves for an extended period of time, provide bleed protection and reduce the need for routine prophylaxis with IV infusions or injections.

Sangamo has had a rough go, cutting its U.S workforce by 40% and streamlining its pipeline last year in an effort to reduce operating expenses. The company did have some good news earlier this month, entering into a potential $1.3 billion licensing deal with Astellas to utilize Sangamo’s proprietary neurotropic adeno-associated virus capsid, STAC-BBB, for one target, with the right to add up to four additional targets, to deliver intravenously administered genomic medicines to treat neurological diseases.