The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.
“What people are looking for with genetic therapies is, could we have a definitive treatment, a single infusion, that would result in steady state expression of factor VIII or factor IX levels that would preclude the need for prophylactic therapies and would prevent all breakthrough bleeding as well? If we can achieve that, can we do it with a good enough safety record to make it amenable to a broad population of patients?”
While gene therapy research in hemophilia has been ongoing for years, the competition is heating up as the condition is a growing indication for both cell and gene therapies. Multiple companies are developing such therapies to address both Factor VIII deficiency in hemophilia A and Factor IX deficiency in hemophilia B.
CGTLive had the opportunity to speak with a veteran of the field, Steven W. Pipe, MD, director, Pediatric Hemophilia and Coagulation Disorders Program, CS Mott Children’s Hospital, and professor, University of Michigan Health, who has been actively involved in research and clinical trials with gene therapies for both hemophilia A and B.
Pipe coauthored a paper published in January 2022 exploring novel approaches to treating hemophilia A and has served as an investigator on the pivotal GENEr8-1 (NCT03370913) study of valoctocogene roxaparvovec (val-rox; Roctavian; BioMarin) as well as other programs by UniQure and Bayer. In this clip, Pipe discussed the unmet needs in the field with current factor replacement therapies and the advantages of adeno-associated virus-based therapies.