List of gene therapies

This article contains a list of commercially available gene therapies.

Gene therapies

See also: Gene therapy § List of gene therapies for treatment of disease

• Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available)
• Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma ^[1]
• Beremagene geperpavec (Vyjuvek): treatment of wounds. ^[2]
• Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia ^[3]
• Brexucabtagene autoleucel (Tecartus): treatment for mantle cell lymphoma and acute lymphoblastic leukemia ^{[4] [5]}
• Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor peripheral artery disease
• Ciltacabtagene autoleucel (Carvykti): treatment for multiple myeloma ^[6]
• Delandistrogene moxeparvovec (Elevidys): treatment for Duchenne muscular dystrophy ^{[7] [8]}
• Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy ^[9]
• Etranacogene dezaparvovec (Hemgenix): AAV-based treatment for hemophilia B ^[10]
• Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. ^[11]
• Gendicine: treatment for head and neck squamous cell carcinoma
• Idecabtagene vicleucel (Abecma): treatment for multiple myeloma ^[12]
• Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. ^[11]
• Nadofaragene firadenovec (Adstiladrin): treatment for bladder cancer ^[13]
• Obecabtagene autoleucel (Aucatzyl): treatment of acute lymphoblastic leukemia ^{[14] [15]}
• Onasemnogene abeparvovec (Zolgensma): AAV-based treatment for spinal muscular atrophy ^[16]
• Revakinagene taroretcel (Encelto): treatment of macular telangiectasia type 2 ^[17]
• Strimvelis: treatment for adenosine deaminase deficiency (ADA-SCID)
• Talimogene laherparepvec (Imlygic): treatment for melanoma in people who have recurring skin lesions ^[18]
• Tisagenlecleucel (Kymriah): treatment for B cell lymphoblastic leukemia ^[19]
• Valoctocogene roxaparvovec (Roctavian): treatment for hemophilia A ^{[20] [21] [22]}
• Voretigene neparvovec (Luxturna): AAV-based treatment for Leber congenital amaurosis ^[23]

See also

• FDA-approved CAR T cell therapies

References

1. "FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma" (Press release). U.S. Food and Drug Administration (FDA). 24 March 2020. Retrieved 16 December 2022.
2. "FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa". U.S. Food and Drug Administration (FDA) (Press release). 19 May 2023. Retrieved 28 May 2023.
3. "FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions" (Press release). U.S. Food and Drug Administration (FDA). 17 August 2022. Retrieved 16 December 2022.
4. "FDA Approves First Cell-Based Gene Therapy For Adult Patients with Relapsed or Refractory MCL" (Press release). U.S. Food and Drug Administration (FDA). 24 July 2020. Retrieved 24 July 2020.
5. "FDA approves brexucabtagene autoleucel for relapsed or refractory B-cell precursor acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 1 October 2021. Retrieved 2 October 2021.
6. "FDA approves ciltacabtagene autoleucel for relapsed or refractory multiple myeloma". U.S. Food and Drug Administration (FDA). 7 March 2022. Retrieved 16 March 2022.
7. "FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy" (Press release). U.S. Food and Drug Administration (FDA). 22 June 2023. Retrieved 22 June 2023.
8. "Sarepta Therapeutics Announces FDA Approval of Elevidys, the First Gene Therapy to Treat Duchenne Muscular Dystrophy" (Press release). Sarepta Therapeutics. 22 June 2023. Retrieved 22 June 2023– via Business Wire.
9. "Skysona". U.S. Food and Drug Administration (FDA). 24 October 2022. Retrieved 16 December 2022.
10. "FDA Approves First Gene Therapy to Treat Adults with Hemophilia B" (Press release). U.S. Food and Drug Administration (FDA). 22 November 2022. Retrieved 16 December 2022.
11. "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease". U.S. Food and Drug Administration (FDA) (Press release). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 8 December 2023.
12. "Abecma". U.S. Food and Drug Administration (FDA). 21 April 2021. Retrieved 16 December 2022.
13. "FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer" (Press release). U.S. Food and Drug Administration (FDA). 16 December 2022. Retrieved 16 December 2022.
14. "FDA approves obecabtagene autoleucel for acute lymphoblastic leukemia". U.S. Food and Drug Administration (FDA). 8 November 2024. Retrieved 10 November 2024.
15. "Autolus Therapeutics Announces FDA Approval of Aucatzyl (obecabtagene autoleucel – obe-cel) for adults with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)" (Press release). Autolus Therapeutics. 8 November 2024. Retrieved 10 November 2024– via GlobeNewswire.
16. "FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality" (Press release). U.S. Food and Drug Administration (FDA). 24 March 2020. Retrieved 16 December 2022.
17. "Encelto". U.S. Food and Drug Administration. 1 October 2024. Retrieved 7 March 2025.
18. "Imlygic (talimogene laherparepvec)". U.S. Food and Drug Administration (FDA). 6 July 2022. Retrieved 16 December 2022.
19. "FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome". U.S. Food and Drug Administration (FDA). 30 August 2017. Retrieved 16 December 2022.
20. "First gene therapy to treat severe haemophilia A" (Press release). European Medicines Agency (EMA). 24 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
21. "Roctavian: Pending EC decision". European Medicines Agency (EMA). 23 June 2022. Archived from the original on 26 June 2022. Retrieved 26 June 2022.
22. "Roctavian". Union Register of medicinal products. 25 August 2022. Retrieved 6 September 2022.
23. "FDA approves novel gene therapy to treat people with a rare form of inherited vision loss". U.S. Food and Drug Administration (Press release). 24 March 2020. Retrieved 16 December 2022.

External links

• Daley, Jim (1 January 2020). "Gene Therapy Arrives". Scientific American. Retrieved 9 June 2020.