Medical technology3 min read
Gene therapy
Gene therapy modifies a person’s genes to treat or cure disease. It is approved today for a small number of conditions — including an inherited eye disorder, spinal muscular atrophy and sickle cell disease — and is being studied for others.
In one approach, cells are taken from the patient and genetically modified (often with a viral vector), then returned to them. In December 2023 the FDA approved the first two cell-based gene therapies for sickle cell disease, and the FDA publishes an updated list of licensed cellular and gene therapy products.
The earliest studies showed that gene therapy can carry serious health risks such as toxicity, and because its techniques are relatively new, some of its risks may be unexpected. For example, the label of one of the two sickle cell therapies approved in 2023 mentions that blood cancer occurred in some people treated with it.
- In widespread clinical use
- Approved for specific uses
- In clinical trials
- Preclinical
- Concept
Specific products are FDA-licensed for a small number of diseases, and other products are still under study.
- Specific genetic diseasesApproved for specific uses
It is used for a small number of diseases including the eye disorder known as Leber congenital amaurosis, and spinal muscular atrophy.2
- Sickle cell diseaseApproved for specific uses
In December 2023 the FDA approved the first two cell-based gene therapies for this disease.3
- Cancer, infectious diseases and other genetic diseasesIn clinical trials
Gene therapy products for these diseases are under study.4
Status last checked: · What the maturity levels mean
What is it?
Gene therapy is a technology that modifies a person's genes to treat or cure a disease.
How does it work?
In one approach, cells are taken from the patient and genetically modified, often using a viral vector, then returned to them.
Newer tools include gene editing, which cuts DNA at a specific location.
Where is it used today?
It is used for a small number of diseases, including an eye disorder and spinal muscular atrophy.
In December 2023 the FDA approved the first two cell-based gene therapies for sickle cell disease.
The FDA publishes an updated list of licensed cellular and gene therapy products.
Limits and risks
The earliest studies showed serious risks such as toxicity, and because the techniques are relatively new, some risks may be unexpected.
The FDA notes that blood cancer occurred in some people treated with one of the two sickle cell therapies approved in 2023.
Common questions
Questions for your doctor
- Is there an approved gene therapy or a clinical trial for my condition?
- What are the known and unknown risks of this therapy?
References
- 1U.S. FDA. Approved Cellular and Gene Therapy Products. www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-productsHealth agencies & guidelines · Accessed 2026-10-08
- 2MedlinePlus Genetics (NLM). What is gene therapy?. medlineplus.gov/genetics/understanding/therapy/genetherapy/Health agencies & guidelines · Accessed 2026-10-08
- 3U.S. FDA. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-diseaseHealth agencies & guidelines · Accessed 2026-10-08
- 4U.S. FDA. What is Gene Therapy?. www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapyHealth agencies & guidelines · Accessed 2026-10-08
- 5NHGRI (NIH). What is genome editing?. www.genome.gov/about-genomics/policy-issues/what-is-Genome-EditingHealth agencies & guidelines · Accessed 2026-10-08
- 6MedlinePlus Genetics (NLM). Is gene therapy safe?. medlineplus.gov/genetics/understanding/therapy/safety/Health agencies & guidelines · Accessed 2026-10-08
Review status: Edited content · Last updated:
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Educational content only — no diagnosis, and no substitute for a clinician.