Nigel Key Co-Authors Pivotal Hemophilia Gene Therapy Study in NEJM

Nigel Key Co-Authors Pivotal Hemophilia Gene Therapy Study in NEJM

Nigel Key was a contributing author on the final analysis of the HOPE-B study results that were published online in in the New England Journal of Medicine on December 7, 2025 coincident with presentation of the data at the ASH meeting in Orlando.

https://www.nejm.org/doi/full/10.1056/NEJMoa2514332?query=RP

The study recruited patients with severe hemophilia B in a phase 3, open label, international protocol. The mean FIX activity attained was 36% with levels remaining constant over 5 years. This pivotal study led to FDA licensure of the first gene therapy protocol for hemophilia