Gene Therapy Today

Examples of FDA-Approved Gene Therapies

After a long history of research, gene therapy is now being used in clinical practice. In this part of genehome, see what gene therapies are approved for use in the United States.
Gene therapies available in the US
Examples of FDA-Approved Gene and Car T Therapies
Gene therapies available in the US
Examples of FDA-Approved Gene and Car T 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, for example, 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 June 2021, the FDA had approved 2 gene therapy products.4

Examples of FDA-approved gene and CAR T therapies

APPROVED GENE THERAPIES
 Type of Therapy  Disease State(s)  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(s)  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 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
Retroviral vector, ex vivo Mantle cell lymphoma (a rare form of non-Hodgkin lymphoma)8  2020
Retroviral vector, ex vivo Certain types of large B-cell lymphoma9  2021
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
Retroviral vector, ex vivo Mantle cell lymphoma (a rare form of non-Hodgkin lymphoma)8  2020
Retroviral vector, ex vivo Certain types of large B-cell lymphoma9  2021
*These are not exhaustive lists of therapies.
To date, the FDA has received more than 900 applications to investigate gene therapy in clinical trials.

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:

  1. Scientists discover the genetic basis of a disease, which might be a mutation in a single gene10
  2. Scientists target their research on ways to modify the specific mutation10
  3. 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 disease11
  4. 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 the safety and efficacy of the treatment11
  5. Successful completion of clinical trials may result in approval by regulatory authorities such as European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) and a specific gene therapy becoming available for people with the disease12,13

To learn more about the impact of regulatory authorities on the history of gene therapy, click here.

Other topics you may be interested in:

Evolution of Gene Therapy

Clinical Trials for Gene Therapy

Resource Center

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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 July 1, 2021. 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 July 1, 2021. https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-products 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. National Cancer Institute. CAR T-Cell therapy approved by FDA for mantle cell lymphoma. Accessed April 28, 2021. https://www.cancer.gov/news-events/cancer-currents-blog/2020/fda-brexucabtagene-mantle-cell-lymphoma 9. Food and Drug Administration. FDA grants accelerated approval to loncastuximab tesirine-lpyl for large B-cell lymphoma. Accessed April 28, 2021. https://www.fda.gov/drugs/fda-grants-accelerated-approval-loncastuximab-tesirine-lpyl-large-b-cell-lymphoma 10. Amberger JS, Bocchini CA, Scott AF, Hamosh A. OMIM.org: leveraging knowledge across phenotype-gene relationships. Nucleic Acids Res. 2019;47(D1):D1038–D1043. 11. Food and Drug Administration. Human gene therapy for rare diseases: guidance for industry. Accessed July 1, 2021. https://www.fda.gov/media/113807/download 12. Food and Drug Administration. Development & approval process: drugs. Accessed July 1, 2021. https://www.fda.gov/drugs/development-approval-process-drugs 13. European Medicines Agency. From laboratory to patient: the journey of a medicine assessed by EMA. Accessed July 1, 2021. https://www.ema.europa.eu/en/documents/other/laboratory-patient-journey-centrally-authorised-medicine_en.pdf

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