Gene therapies are being developed to treat many types of genetic diseases, including those that affect the neuromuscular system, central nervous system, liver, eyes, and other tissues. Gene therapy can target specific genes that lead to disease by helping to replace, bypass, or silence the gene that isn't working properly.
How genes work
Know the basics of genetics to help you understand the role genes play in inherited diseases.
How gene therapy works
Learn how gene therapy works to regulate the way a gene functions, so the cell can produce better functioning proteins
How gene therapy is used
See how gene therapies are being used to treat rare inherited diseases
Additional resources
Find additional resources and information about organizations offering support.
Be equipped with questions to ask your doctor about clinical trials in gene therapy.
Additional resources and support
Doctor Discussion Guide
US Food and Drug
Administration
(FDA) approvals
for gene
therapies
through February 20231
Clinical trials
conducted or ongoing worldwide
as of February 20202
Years of
scientific research3
References
1. Approved cellular and gene therapy products. US Food and Drug Administration. Updated December 16, 2022. Accessed February 21, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products 2. Maldonado R, Jalil S, Wartiovaara K. J Community Genet. 2021;12:267-276. 3. Asher DR, et al. Clinical development on the frontier: Gene therapy for Duchenne muscular dystrophy. Expert Opin Biol Ther. 2020;20:263-274.
Astellas Gene Therapies is not responsible for the content provided by third-party sites or organizations, nor does Astellas Gene Therapies endorse the content of these sites or organizations.
