Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B

June 12, 2025

CONCLUSIONS

A single administration of scAAV2/8-LP1-hFIXco gene therapy resulted in durable fac-

tor IX expression, sustained clinical benefit, and no late-onset safety concerns over a

period of 13 years. These data support the long-term efficacy and safety of AAV gene

therapy for severe hemophilia B. (Funded by the U.K. Medical Research Council and

others; ClinicalTrials.gov number, NCT00979238; EudraCT number, 2005-005711-17.)