Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia


The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.

“As it stands right now, the safety and efficacy look quite encouraging. We believe that at least 1 platform for hemophilia A and 1 for hemophilia B will be submitted for regulatory review this year. I'm fully preparing our clinic to the fact that we may be doing commercial gene therapy either late in 2022 or early in 2023.”

Multiple companies are targeting hemophilia A and B indications for gene and cell therapy programs, with the goal of addressing 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 who has been involved with multiple gene therapy investigations. Pipe is 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.

Among Pipe’s investigations are the pivotal GENEr8-1 (NCT03370913) study of valoctocogene roxaparvovec (val-rox; Roctavian; BioMarin), the BLA filing for which was recently delayed by the FDA, as well as studies on etranacogene dezaparvovec by UniQure and another program by Bayer.1 A paper coauthored by Pipe was published in January 2022 exploring novel approaches to treating hemophilia A.2 In this clip, Pipe discussed the challenges of conducting gene therapy investigations in hemophilia.

1. BioMarin delays hemophilia a therapy valoctocogene roxaparvovec filing as FDA calls for more data. News release. BioMarin. May 31, 2022.
2. 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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