Longevity of hemophilia gene therapy in question as BioMarin seeks accelerated approval

As BioMarin reported interim Phase III data Tuesday that sets the company up for regulatory submissions, long-term Phase I/II data cast doubt on the durability of gene therapy valoctocogene roxaparvovec to treat severe hemophilia A. The product is an adeno-associated viral serotype 5 (AAV5) vector delivering the Factor VIII gene.

Among 16 evaluable patients treated with a single

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