The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed current studies evaluating gene therapies in hemophilia.
“What we're gearing up for this year is how to prepare the hemophilia treatment centers to roll out and maintain gene therapy programs with good safety and outcomes for patients in the commercial setting. So, we'll be getting sites ready and patients ready to embrace this technology, if it's right for them.”
Hemophilia A and B are growing 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 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.
Pipe has been involved with multiple gene therapy investigations. Among his 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.1 He is also involved in studies on etranacogene dezaparvovec by UniQure and another program by Bayer and recently coauthored a paper exploring novel approaches to treating hemophilia A.2