Guess the Therapy Answer February 18, 2026

Which gene therapy developed by CSL Behring and uniQure was approved by the FDA for hemophilia B in 2022?

Answer: etranacogene dezaparvovec (marketed as Hemgenix)

See below for further reading on Hemgenix:

Integrating Gene Therapy into Clinical Care for Hemophilia B

At the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, end-of-study data were presented from the pivotal phase 3 HOPE-B clinical trial (NCT03569891) that supported the FDA’s 2022 approval of CSL Behring/uniQure’s Hemgenix, which is an FDA-approved adeno-associated virus (AAV) vector-based gene therapy intended to treat hemophilia B. At the conference CGTLive® interviewed HOPE-B principal investigator Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health.

After asking Pipe about the findings and their implications, CGTLive® asked Pipe more generally about what clinicians thinking about incorporating the commercial product into their practice need to know. Pipe went over key considerations for infrastructure, speaking to patients about the treatment decision, and more.

Steven W. Pipe, MD, on the End-of-Study Results From Hemgenix’s HOPE-B Trial

At the 67th ASH Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, end-of-study data from HOPE-B were presented by Pipe, who served as the trial’s principal investigator. CGTLive® sat down with Pipe at the conference to learn more about the updated findings. Pipe went over the key efficacy and safety data, noting that of 54 participants, only 2 did not respond to the treatment. The remaining patients showed stable factor IX activity, averaging 36% at 5 years after treatment with Hemgenix. Furthermore, annualized bleeding rates fell sharply, dropping from about 4 per year at baseline to 1.5 per year across follow up. Pipe also noted that safety was favorable, with the vast majority of treatment-related adverse events occurring in the first 6 months.