Gene Therapy Today
Examples of Approved Gene Therapies


Gene therapies available in the US
Since the beginning of gene therapy research over 40 years ago, our understanding and technological advancements in gene therapy have taken giant leaps forward.1 In 2017, after extensive research in labs and in human clinical trials around the world, the first gene therapies were approved by the Food and Drug Administration (FDA) for use in the United States.2,3
As of January 2020, the FDA had approved 2 gene therapy products.4
APPROVED GENE THERAPIES | ||
Type of Therapy | Disease State | Year Approved |
Gene Addition | ||
Adeno-associated virus vector, in vivo | Inherited retinal dystrophy5 | 2017 |
Adeno-associated virus vector, in vivo | Spinal muscular atrophy6 | 2019 |
APPROVED GENE THERAPIES | ||
Type of Therapy | Disease State | Year Approved |
Gene Addition | ||
Adeno-associated virus vector, in vivo | Inherited retinal dystrophy5 | 2017 |
Adeno-associated virus vector, in vivo | Spinal muscular atrophy6 | 2019 |
Additionally, two gene based cellular immunotherapies were approved by FDA as of Jan 2020.
APPROVED CAR T-CELL THERAPIES | ||
Type of Therapy | Disease State | Year Approved |
CAR T | ||
Lentiviral vector, ex vivo | Acute lymphoblastic leukemia (ALL)7 | 2017 |
Retroviral vector, ex vivo | Relapsed or refractory large B-cell lymphoma3 | 2017 |
APPROVED CAR T-CELL THERAPIES | ||
Type of Therapy | Disease State | Year Approved |
CAR T | ||
Lentiviral vector, ex vivo | Acute lymphoblastic leukemia (ALL)7 | 2017 |
Retroviral vector, ex vivo | Relapsed or refractory large B-cell lymphoma3 | 2017 |

Gene-ius Questions
To help understand gene therapy, here is a general example of a gene therapy and how it moves from research to a potential therapeutic reality:
- Scientists discover the genetic basis of a disease, which might be a mutation in a single gene8
- Scientists target their research on ways to modify the specific mutation8
- Different kinds of gene therapy techniques are explored in a laboratory in order to figure out which are most effective and show the most promise in treating a genetic disease9
- Once scientists find a technique that they believe has the potential to work, the gene therapy goes through preclinical research and rigorous clinical trials to evaluate safety and efficacy of the treatment9
- Successful completion of clinical trials may result in approval by the European Medicines Agency (EMA) and/or the US Food and Drug Administration (FDA) and a specific gene therapy becoming available to people with the disease10,11
Other topics you may be interested in:

Sign up for updates
References
1. Wirth T, Parker N, Ylä-Hertuala. History of gene therapy. Gene. 2013;525(2):162-169. 2. Food and Drug Administration. FDA approval brings first gene therapy to the United States. Press release. Accessed March 4, 2020. https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states 3. Yescarta® (axicabtagene ciloleucel) [prescribing information]. Santa Monica, CA: Kite Pharma, Inc.; 2017. 4. Food and Drug Administration. FDA continues strong support of innovation in development of gene therapy products. Press release. Accessed March 4, 2020. https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-productsc 5. Luxturna® (voretigene neparvovec-rzyl) [prescribing information]. Philadelphia, PA: Spark Therapeutics, Inc.; 2017. 6. Zolgensma® (onasemnogene abeparvovec-xioi) [prescribing information]. Bannockburn, IL: AveXis, Inc.; 2019. 7. Kymriah® (tisagenlecleucel) [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals; 2017. 8. Amberger JS, Bocchini CA, Scott AF, Hamosh A. OMIM.org: leveraging knowledge across phenotype-gene relationships. Nucleic Acids Res. 2019;47(D1):D1038–D1043. 9. Food and Drug Administration. Human gene therapy for rare diseases: guidance for industry. Accessed March 4, 2020. https://www.fda.gov/media/113807/download 10. Food and Drug Administration. Development & approval process: drugs. Accessed March 4, 2020. https://www.fda.gov/drugs/development-approval-process-drugs 11. European Medicines Agency. From laboratory to patient: the journey of a medicine assessed by EMA. Accessed March 4, 2020. https://www.ema.europa.eu/en/documents/other/laboratory-patient-journey-centrally-authorised-medicine_en.pdf