ASGCT Briefs Capitol Hill on Gene Therapy Breakthroughs for Hemophilia

On July 24, ASGCT held an educational briefing to inform legislators and staff about the impact of innovative hemophilia gene therapies. This is the latest in a series of briefings on CAR T, gene editing, and sickle cell disease.  

Hemophilia, a genetic disorder characterized by chronic impaired blood clotting, has long posed significant challenges to patients and healthcare systems. Traditional treatments involve regular and lifelong infusions of clotting factor products, which can be burdensome and may be only partially effective. The advent of gene therapies is changing the hemophilia landscape, offering the potential for long-lasting, durable treatments.  

Gene therapies work by addressing the root cause of hemophilia—mutations in the genes responsible for clotting factor production. By delivering functional copies of these genes to patients' cells, gene therapies can enable the production of clotting factors within the body, significantly reducing or even eliminating the need for regular infusions. This shift not only improves health outcomes but also dramatically enhances the quality of life for individuals with hemophilia. Patients can experience fewer bleeding episodes, reduced joint damage, and greater overall well-being. 

During the briefing, experts highlighted the real-world benefits of gene therapy for hemophilia patients. The briefing provided a comprehensive overview of the gene therapy landscape, from basic mechanisms and delivery methods, to clinical results and commercialization. Patient advocates also shared their experience receiving gene therapy for hemophilia B, highlighting their lifelong clinical journey with the disease, the daily changes they’ve seen post-gene therapy treatment, and the importance of support structures in their treatment journey. The advocates explained their different gene therapy treatments for hemophilia B (HEMGENIX and BEQVEZ), and their separate paths to obtaining treatment through clinical trials nearly 6 years ago. ASGCT thanks Matt Porteus, MD, PhD; Lindsey George, MD; Len Valentino, MD; Bobby Wiseman; and Bill Maurits for sharing their expertise and lived experiences.  

With three gene therapy products already approved by FDA - one for hemophilia A and two for hemophilia B - and more in the pipeline, it is essential for policymakers to understand both the potential and the challenges of these therapies. ASGCT aims to foster a supportive environment for the continued development and implementation of gene therapies. Ensuring that patients can access these therapies requires thoughtful consideration of insurance coverage, manufacturing challenges, and the integration of new treatment paradigms into existing healthcare frameworks. 

 ASGCT remains committed to advocating for policies that support innovation and patient access to improve the lives of those affected by hemophilia and other genetic disorders. 

Christina is ASGCT's Senior Manager of Government Affairs.

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