The “Top 10” list of best-selling gene therapies published by Genetic Engineering & Biotechnology News (GEN) in their May 2025 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:

Ranked Number 6:    Roctavian® (valoctocogene roxaparvovec-rvox; “val-rox”l) from BioMarin Pharmaceutical

                                  Roctavian is 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.

                                  FDA approval: June 30, 2023

Ranked Number 8:    Luxturna® (voretigene neparvovec-rzyl) from Spart Therapeutics (Roche) 

                                  Luxturna is 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.

                                  FDA approval: March 26, 2021

Noted but unranked:    Hemgenix® (etranacogene dezaparoavec) from uniQure /CSL Behring

                                     Hemgenix is an AAV vector-based gene therapy indicated for adults with hemophilia B (congenital factor IX   deficiency based on technology licensed from the NHLBI and NIDCR.

                                     FDA approval: November 22, 2022

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