Hemgenix®
Company
uniQure & CSL
Use
Treatment of adults with hemophilia B.
Features
A one-time gene therapy for hemophilia B that allows long-term expression of Factor IX. FDA approved in 2022.
NIH Contribution
NIH research on gene therapy methods.
HHS Institute/Agency
NHLBI
Inventor(s)
Chiorini, J. et al.
Year
2023
