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 May 2023.2 Please see FDA website for full indications of therapies listed.

August 2017: tisagenlecleucel

For B-cell precursor acute lymphoblastic leukemia
(Approved May 2018 for relapsed or refractory large B-cell lymphoma)

October 2017: axicabtagene ciloleucel

For relapsed or refractory large B-cell lymphoma
(Approved April 2021 for relapsed or refractory follicular lymphoma)

December 2017: voretigene neparvovec-rzyl

For inherited retinal disease

May 2019: onasemnogene abeparvovec

For spinal muscular atrophy (type I)

July 2020: brexucabtagene autoleucel

For relapsed or refractory mantle cell lymphoma
(Approved October 2021 for relapsed or refractory B-cell precursor acute lymphoblastic leukemia)

February 2021: lisocabtagene maraleucel

For relapsed or refractory large B-cell lymphoma

March 2021: idecabtagene vicleucel

For relapsed or refractory multiple myeloma after four or more prior lines of therapy

August 2022: betibeglogene autotemcel

For β-thalassemia requiring regular red blood cell (RBC) transfusion

November 2022: etranacogene dezaparvovec-drlb

For hemophilia B (congenital Factor IX deficiency)

December 2022: nadofaragene firadenovec-vncg

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

February 2023: ciltacabtagene autoleucel

For relapsed or refractory multiple myeloma

April 2023: omidubicel-onlv

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

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 April 14, 2023. 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 May 19, 2023. Accessed April 14, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.