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Today, CSL Behring announced that the.
If approved, this would be the first gene therapy to treat hemophili.
People with hemophilia B have mutations in the gene encoding coagulation factor IX (FIX), resulting in a loss or deficiency of F.
Transgenes expressing FIX Padua variants were delivered using AAV5 vecto.
The patient's annual bleeding rate (ABR) was 64% lower than in the run-in period before receiving treatment (p=000
References:
References:[1] FDA Accepts CSL Behring's Biologics License Application for Etranacogene Dezaparvovec for Priority Revi.