BioMarin Investigational Gene Therapy Updates

DNA strand

An update for the hemophilia community from BioMarin regarding a previously reported serious adverse event in the ongoing hemophilia A gene therapy [valoctocogene roxaparvovec, BMN 270] Phase 3 study that was deemed, by the Independent Data Safety Monitoring Committee and the company, very unlikely to be related to the investigational therapy. Clinical Development Program Update […]

Pfizer Reports First Quarter 2022 Update of Gene Therapy Trial

Read the original press release. During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the U.S. Food and Drug Administration lifted a clinical hold that had been placed on the Phase 3 AFFINE study in November 2021 following the observance of Factor VIII levels greater than 150% in some study participants. Pfizer […]