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Comparing durability of leading hemophilia A gene therapies

BioMarin, Pfizer-Sangamo products keep patients in the mild disease category for at least two years, though Factor activity wanes

By Danielle Golovin, Staff Writer
January 13, 2022 1:43 AM UTC

New data provide an updated picture about the durability of hemophilia A gene therapies, showing that although Factor VIII activity wanes, the most advanced programs achieve activity levels that keep patients’ disease mild for at least two years. 

On Sunday, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced two-year results from the Phase III GENEr8-1 study of valoctocogene roxaparvovec to treat hemophilia A. ...

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BCIQ Company Profiles

Biomarin Pharmaceutical Inc.

Pfizer Inc.

Sangamo Therapeutics Inc.

BCIQ Target Profiles

Factor VIII