Pfizer Sangamo Breakup Puts China CGT in Spotlight

（the article has been updated to make the text concise and cohesive)

Pfizer’s decision to dissolve its longstanding deal with Sangamo Therapeutics may open new doors for gene therapy (CGT) players in China.

In the final days of 2024, Pfizer ended its collaboration with Sangamo, relinquishing the rights to the gene therapy for Hemophilia A, giroctocogene fitelparvovec, which is currently in Phase 3 development. As a result, Pfizer effectively walked away from filing a biologics license application (BLA) with the FDA and a marketing authorization application (MAA) with the European Union.

This decision likely came as a surprise, sending Sangamo’s stock plummeting by as much as 67% during after-hours trading.

Just weeks prior, on December 9, Pfizer had presented detailed clinical trial data for the gene therapy, AFFINE, at the 66th American Society of Hematology (ASH) Annual Meeting. At that time, the company indicated it was in discussions with regulatory authorities about a potential BLA and MAA, which could have been filed in early 2025.

Sangamo’s CEO, Sandy Macrae, expressed being “surprised and extremely disappointed” by the termination and said the company would seek a new partner to continue development.

In the meantime, competition in gene therapy for Hemophilia A is heating up, particularly from China. One notable contender is Shanghai-based Belief BioMed, which is developing a gene therapy for Hemophilia B, BBM-H901. Belief has already filed a new drug application with China’s National Medical Products Administration (NMPA), which has accepted the application for review.

Belief BioMed has granted commercial rights for BBM-H901 in China to Takeda Pharmaceuticals. The company’s co-founder, Xiao Xiao, a professor at the University of North Carolina, told Yang Report that Belief does not plan to develop or market the gene therapy outside of China.

This approach stems from the fact that many gene therapy developers in China, like Belief, rely on investigator-initiated studies (IIT) to accelerate data collection. This method allows for quicker initiation of studies and treatment of patients, but the resulting data cannot be used for filings outside of China.