Steven W. Pipe, MD, on Integrating Gene Therapy into Clinical Care for Hemophilia B

This is the third part of an interview with Steven W. Pipe, MD. For the second part, click here.

“Even at my center, we've had to think about what sort of infrastructure we need to develop, what kind of education we need to provide for our multidisciplinary staff, what additional staff might we need in order to be able to deliver this effectively—and that's actually been a process over a couple of years to get to the point where we can now actually start to offer this to patients.”

CSL Behring/uniQure’s etranacogene dezaparvovec (marketed as Hemgenix), a gene therapy product for the treatment of severe or moderately severe hemophilia B with or without preexisting AAV5 neutralizing antibodies (NAbs), has been approved by the FDA since 2022.¹ In the time since then, it has begun its rollout into clinical practice for patients with hemophilia B in the United States. In light of this, those working at clinics looking to incorporate the product need to consider several important factors.

At the the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® spoke with Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, who served as the principal investigator for the pivotal phase 3 HOPE-B clinical trial (NCT03569891) that supported Hemgenix’s approval by the FDA. After interviewing him about the final results from HOPE-B that he presented at the conference, CGTLive® asked about practical considerations for the integration of Hemgenix into real-world use.²

Pipe explained that centers must build new infrastructure, educate multidisciplinary staff, and sometimes add personnel, a process that can take years. He emphasized the importance of shared decision-making with patients and families, using long-term efficacy and safety data to compare gene therapy with standard prophylaxis, particularly regarding joint health. Pipe also noted that posttreatment monitoring focuses on liver enzyme surveillance, which can be managed through local labs to reduce patient burden.

For more coverage of ASH 2025, click here.

REFERENCES
1. FDA approves first gene therapy to treat adults with hemophilia B. News release. FDA. November 22, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b
2. Pipe S, Miesbach W, Recht M, et al. End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years. Presented at: ASH 2025 Annual Meeting. December 6-9, 2025; Orlando, FL. Abstract #538