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FDA Clears $2.9M Gene Therapy

The therapy uses an inactivated virus to deliver a replacement gene to liver cells responsible for producing the clotting protein.

Biomarin
Biomarin

Hemophilia A is an inherited blood-clotting disorder that can lead to excessive bleeding, even from minor injuries. According to a recent ABC News article, the FDA has granted approval to BioMarin for its gene therapy called Roctavian, designed to treat severe cases of hemophilia A. Roctavian is an infused treatment that aims to reduce dangerous bleeding problems associated with the condition. It’s the first gene therapy approved for patients with severe hemophilia A.

INTRODUCING! The Latest Trends for Life Sciences at PACK EXPO Southeast
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INTRODUCING! The Latest Trends for Life Sciences at PACK EXPO Southeast