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End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.

U.S. drugmaker Pfizer Inc. said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study.

If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.

Bristol Myers Squibb is terminating a seven-year-old gene therapy collaboration to develop potential treatments for congestive heart failure with uniQure N.V. valued at $1 billion.

BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.

Topline data from Lysogene’s Phase II/III AAVance study showed its investigational gene therapy, LYS-SAF302, failed to meet its primary and key secondary efficacy endpoints in mucopolysaccharidosis type IIIA.