3 Gene Therapies from NIH Make GEN Top 10 Best Sellers List

By: Richelle Holnick, NIH OTT
Originally published in the July 2026 NIH Technology Transfer Community Newsletter.

A "Top 10" list of best-selling gene therapies published by Genetic Engineering & Biotechnology News (GEN) in their May 2026 issue features three products based upon license agreements for technologies originating from the NIH Intramural Research Program.

Featured and ranked on the GEN list are:

• Hemgenix®, a one-time gene therapy for hemophilia B that allows long-term expression of Factor IX. Hemgenix is based on technology licensed from the National Heart, Lung, and Blood Institute and the National Institute of Dental and Craniofacial Research. It received FDA approval in 2022.
• Luxturna®, an adeno-associated virus vector-based gene therapy for treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy based on technology licensed from NEI.
• Roctavian®, an AAV vector-based gene therapy for treatment of adults with severe hemophilia A (congenital factor VIII deficiency) based upon technology licensed from NHLBI and NIDCR.

Congratulations to NIH intramural investigators and their technology transfer programs for such an impressive showing!