Gene-therapy research

Learn about diseases under research and approved treatments

As research in gene therapy continues to advance, many disease applications are actively under clinical investigation and several therapies are already approved by the US Food & Drug Administration (FDA).1,2 Stay up to date so you are prepared to answer gene-therapy questions from patients.

Patient looking hopeful about the future

Gene-therapy research

Learn about diseases under research and approved treatments

As research in gene therapy continues to advance, many disease applications are actively under clinical investigation and several therapies are already approved by the US Food & Drug Administration (FDA).1,2 Stay up to date so you are prepared to answer gene-therapy questions from patients.

Diseases under research for gene therapy

The below list includes just some of the diseases currently in clinical trials for gene therapy.1

Alzheimer disease

β-thalassemia

Blindness

Cancer

Cystic fibrosis

Hemophilia

Hepatitis C

HIV

Huntington disease

Infectious disease

Muscular dystrophy

Parkinson disease

Severe combined immunodeficiency syndrome

Sickle cell disease

Keep up with the latest research in the fast-moving field of gene therapy

View US gene-therapy clinical trials
Lab microscope icon representing gene therapy research

Approved cell and genetic therapies

The below timeline outlines cell and genetic therapies approved by the FDA through October 2024.2 Please see FDA website for full indications of therapies listed. This list is not meant to capture additional approved indications for each therapy listed.

August 2017: tisagenlecleucel

Initially approved for B-cell precursor acute lymphoblastic leukemia

October 2017: axicabtagene ciloleucel

Initially approved for relapsed or refractory large B-cell lymphoma

December 2017: voretigene neparvovec-rzyl

Initially approved for inherited retinal disease

May 2019: onasemnogene abeparvovec-xioi

Initially approved for spinal muscular atrophy

July 2020: brexucabtagene autoleucel

Initially approved for relapsed or refractory mantle cell lymphoma

February 2021: lisocabtagene maraleucel

Initially approved for relapsed or refractory large B-cell lymphoma

March 2021: idecabtagene vicleucel

Initially approved for relapsed or refractory multiple myeloma after four or more prior lines of therapy

February 2022: ciltacabtagene autoleucel

Initially approved for relapsed or refractory multiple myeloma

August 2022: betibeglogene autotemcel

Initially approved for β-thalassemia requiring regular red blood cell (RBC) transfusion

September 2022: elivaldogene autotemcel

Initially approved for early, active cerebral adrenoleukodystrophy (CALD)

November 2022: etranacogene dezaparvovec-drlb

Initially approved for hemophilia B (congenital Factor IX deficiency)

December 2022: nadofaragene firadenovec-vncg

Initially approved for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors

Initially approved for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors

April 2023: omidubicel-onlv

Initially approved for hematologic malignancies that are planned for umbilical cord blood transplantation following myeloablative conditioning

May 2023: beremagene geperpavec

Initially approved for the treatment of wounds in people with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene 

June 2023: donislecel-jujn

Initially approved for type 1 diabetes

June 2023: valoctocogene roxaparvovec-rvox

Initially approved for severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL)

December 2023: exagamglogene autotemcel

Initially approved for people with sickle cell disease who have recurrent vaso-occlusive crises

December 2023: lovotibeglogene autotemcel

Initially approved for people with sickle cell disease who have a history of vaso-occlusive events

March 2024: atidarsagene autotemcel

Initially approved for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD)

April 2024: fidanacogene elaparvovec-dzkt

Initially approved for treating moderate to severe hemophilia B (congenital factor IX deficiency)

June 2024: delandistrogene moxeparvovec-rokl

Initially approved for ambulatory and non-ambulatory pediatric patients with Duchenne muscular dystrophy (DMD)

Discover the history of gene therapy

Check out our gene-therapy timeline to see how far the field has come.

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References: 1. Gene therapy. ClinicalTrials.gov. Accessed September 3, 2024. https://clinicaltrials.gov/ct2/results?cond=&term=gene+therapy&cntry=US&state=&city=&dist. 2. Approved cellular and gene therapy products. US Food & Drug Administration. Updated August 2024. Accessed September 3, 2024. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.