Gene Therapy Potential in Hemophilia: Steven W. Pipe, MD


The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed research with AAV gene therapy in hemophilia.

"It’s the immune response that I think most people are most concerned about as this poses the greatest risk for losing expression over time. So armed with all the information that we learned from the early clinical trials, we got enough evidence of good expression from phase 1 and phase 1 for both hemophilia A and B, that several programs have now moved on to pivotal trials in phase 3.”

Hemophilia is a growing indication for both cell and gene therapies and competition is heating up as multiple companies develop these kinds of therapies to address both Factor VIII deficiency in hemophilia A and Factor IX deficiency in hemophilia B.

CGTLive had the opportunity to speak with Steven W. Pipe, MD, a veteran of the field and gene therapy investigations in the field. He serves as director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital and is also a professor at University of Michigan Health.

Pipe has served as an investigator on the pivotal GENEr8-1 (NCT03370913) study of valoctocogeneroxaparvovec (val-rox; Roctavian; BioMarin) as well as other programs by UniQure and Bayer. He also coauthored a paper published in January 2022 exploring novel approaches to treating hemophilia A. In this clip, Pipe discussed the potential of AAV gene therapy for the treatment of hemophilia as well as challenges that remain with this approach.

Pipe SW, Gonen-Yaacovi G, Segurado OG. Hemophilia A gene therapy: current and next-generation approaches. Expert Opin Biol. Published online January 6, 2022. doi:10.1080/14712598.2022.2002842
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