Gene Therapy Today

Examples of 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

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

To date, the FDA has received more than 900 applications to investigate gene therapy in clinical trials. This new age of medicine can give people whose lives revolve around managing their disease increased therapeutic choices.4
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 gene8
  2. Scientists target their research on ways to modify the specific mutation8
  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 disease9
  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 safety and efficacy of the treatment9
  5. 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

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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 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

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