Roctavian®
Company
BioMarin Pharmaceutical
Use
Treatment of adults with severe hemophilia A.
Features
A gene therapy using adeno-associated virus vector to deliver a functional gene to hemophilia A patients. FDA approved in 2023.
NIH Contribution
Provided patented technologies related to gene therapy vectors.
HHS Institute/Agency
NHLBI
Inventor(s)
Kotin, R. et al.
Year
2023
