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Hemophilia A Gene Therapy Race Intensifies With Updated Sangamo/Pfizer Data

 
• By Jessica Merrill

Sangamo and Pfizer released positive Phase I/II data from a cohort of patients treated with a higher dose of the gene therapy SB-525 for hemophilia A, but rivals are further ahead in development.

GeneTherapy

Positive early data on Sangamo Therapeutics Inc./Pfizer Inc.'s gene therapy for hemophilia A at a higher dose cohort have put momentum behind the program, but the partners are up against rivals BioMarin Pharmaceutical Inc. and Spark Therapeutics Inc., two companies that are further ahead in the race to bring the first gene therapy for the blood disorder to the market.

Pfizer and Sangamo reported interim data April 2 from the Phase I/II Alta study, showing SB-525 was well tolerated and demonstrated a dose-dependent increase in Factor VIII levels across four dosage cohorts. A safety monitoring committee recommended the high dose cohort – 3e13 vg/kg – be expanded

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