⁤BioMarin has decided to discontinue its preclinical gene therapy, known as BMN 293, which aims to treat myosin-binding protein C3 (MYBPC3) hypertrophic cardiomyopathy, a condition that thickens heart muscles. ⁤⁤Despite promising preclinical results BioMarin determined that the time and resources required for BMN 293’s development no longer aligned with its strategic priorities. ⁤⁤This decision follows BioMarin’s earlier pipeline reduction in April, where it halted development on several other therapies. ⁤⁤BioMarin is now concentrating its efforts on three key candidates: BMN 351 for Duchenne muscular dystrophy, BMN 349 for Alpha-1 antitrypsin deficiency-associated liver disease, and BMN 333 for growth disorders. ⁤⁤Additionally, the company announced a more targeted rollout of its hemophilia A gene therapy, Roctavian, focusing solely on the U.S., Germany, and Italy to improve profitability. ⁤⁤Despite slow uptake, Roctavian generated $7 million in revenue in the second quarter of 2024.

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[Source: Fierce Biotech, August 6th 2024.]