The “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:

In sixth place is 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 NHLBI and NIDCR. It received FDA approval in 2022. Hemgenix was noted but unranked last year. 

In eight place is 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. 

In tenth place is 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 our intramural investigators and their technology transfer programs for such an impressive showing!