Factor Ix

A single infusion of verbrinacogene setparvovec resulted in normal factor IX levels among all 10 men who received the investigational gene therapy for moderate to severe hemophilia B, study results showed.

The novel gene therapy BAX 335 did not induce sustained factor IX expression among patients with hemophilia B, according to an interim analysis of a phase 1/phase 2 dose-escalation study published in Blood.

ORLANDO, Fla. — In this video at the ASH Annual Meeting and Exposition, Steven W. Pipe, MD, professor of pediatrics and pathology at the University of Michigan, reviews recent innovations in hemophilia and incorporating gene therapy into practice.