History of Gene Therapy
The exciting field of gene therapy 50 years in the making.
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References
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- The New York Times, September 21, 1970;
- Terheggen HG, et al. Z Kinderheilkd 1975;119(1):1–3;
- Friedmann T, Roblin R. Science 1972;175(4025):949–955.
- Rosenberg SA, et al. N Engl J Med 1990;323(9):570–578.
- Wirth T, et al. Gene 2013;525(2):162–169;
- Blaese RM, et al. Science 1995;270(5235):475–480;
- Dunbar CE, et al. Science 2018;12;359(6372). pii:eaan4672;
- NHS. Severe combined immunodeficiency (SCID). Available at: https://www.gosh.nhs.uk/conditions-and-treatments/conditions-we-treat/severe-combined-immunodeficiency-scid. Accessed October 4, 2019.
- Verma IM. Mol Ther 2000;2(5):415–416;
- Sibbald B. CMAJ 2001;164(11):1612;
- Lehrman S. Nature 1999;401(6753):517–518.
- Biopharm International. The Genesis of Gendicine: The Story Behind the First Gene Therapy. Available at: http://www.biopharminternational.com/genesis-gendicine-story-behind-first-gene-therapy. Accessed October 4, 2019;
- Zhang WW, et al. Hum Gene Ther 2018;29(2):160–179;
- Zhang S-Y, et al. Chapter 10 - Recombinant adenoviral-p53 agent (Gendicine®): Quality control, mechanism of action, and its use for treatment of malignant tumors. In: Liu XX, et al (eds). Recent Advances in Cancer Research and Therapy. Elsevier, 2012:215–243. Available at: https://www.sciencedirect.com/science/article/pii/B9780123978332000108. Accessed October 4, 2019;
- Peng Z. Hum Gene Ther 2005;16(9):1016–1027.
- Yu TTL, et al. Front Bioeng Biotechnol 2018;6:130;
- EMA. Press release. March 11, 2010. Available at: https://www.ema.europa.eu/en/documents/press-release/ark-therapeutics-ltd-withdraws-its-marketing-authorisation-application-cerepro-sitimagene_en.pdf. Accessed October 4, 2019.
- European Commission. Glybera. Available at: https://ec.europa.eu/health/documents/community-register/html/h791.htm. Accessed October 4, 2012
- EMA. Press release. July 20, 2012. Available at: https://www.ema.europa.eu/en/news/european-medicines-agency-recommends-first-gene-therapy-approval. Accessed October 4, 2019;
- EMA. EPAR summary for the public. Glybera. October 2015. Available at: https://www.ema.europa.eu/documents/overview/glybera-epar-summary-public_en.pdf. Accessed October 4, 2019;
- Glybera [summary of product characteristics – medicinal product no longer authorized]. 2017. Available at: https://www.ema.europa.eu/en/documents/product-information/glybera-epar-product-information_en.pdf. Accessed October 4, 2019;
- uniQure. Press release. April 20, 2017. Available at: http://uniqure.com/GL_PR_Glybera%20withdrawal_FINAL_PDF.pdf. Accessed October 4, 2019;
- PharmaPhorum. Press release. April 20, 2017. Available at: https://pharmaphorum.com/news/glybera-expensive-drug-world-withdrawn-commercial-flop/. Accessed October 4, 2019.
- European Commission. Strimvelis. Available at: https://ec.europa.eu/health/documents/community-register/html/h1097.htm. Accessed October 4, 2019;
- GSK. Press release. May 27, 2016. Available at: https://www.gsk.com/en-gb/media/press-releases/strimvelistm-receives-european-marketing-authorisation-to-treat-very-rare-disease-ada-scid/. Accessed October 4, 2019;
- Strimvelis® [summary of product characteristics]. 2018. Available at: https://www.ema.europa.eu/en/documents/product-information/strimvelis-epar-product-information_en.pdf. Accessed October 4, 2019;
- Hoggatt J. Cell 2016;166(2):263.
- U.S. FDA. News release. August 30, 2017. Available at: https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm574154.htm. Accessed August 20, 2018;
- Kymriah® [package insert]. 2018. Available at: https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymriah.pdf. Accessed October 4, 2019.
- U.S. FDA. News release. October 18, 2017. Available at: https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm581296.htm. Accessed October 4, 2019;
- Yescarta® [package insert]. 2017. Available at: https://www.fda.gov/media/108377/download. Accessed October 4, 2019.
- Luxturna™ [package insert]. 2017. Available at: http://sparktx.com/LUXTURNA_US_Prescribing_Information.pdf. Accessed October 4, 2019;
- U.S. FDA. News release. December 18, 2017. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm. Accessed October 4, 2019;
- Wang D, et al. Nat Rev Drug Discov 2019;18:358–378.
- U.S. FDA. News release. May 1, 2018. Available at: https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm606540.htm. Accessed August 20, 2019.
- European Commission. Kymriah. Available at: https://ec.europa.eu/health/documents/community-register/html/h1297.htm. Accessed October 4, 2019;
- European Commission. Yescarta. Available at: https://ec.europa.eu/health/documents/community-register/html/h1299.htm. Accessed October 4, 2019;
- Yescarta® [summary of product characteristics]. 2018. Available at: https://www.ema.europa.eu/en/documents/product-information/yescarta-epar-product-information_en.pdf. Accessed October 4, 2019.
- European Commission. Luxturna. Available at: https://ec.europa.eu/health/documents/community-register/html/h1331.htm. Accessed October 4, 2019;
- EMA. Luxturna. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/luxturna#authorisation-details-section. Accessed October 4, 2019;
- Spark Therapeutics. Press release. November 23, 2018. Available at: http://ir.sparktx.com/news-releases/news-release-details/european-commission-approves-spark-therapeutics-luxturnar. Accessed October 4, 2019.
- U.S. FDA. News release. May 24, 2019. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. Accessed October 4, 2019;
- Mendell JR, et al. N Engl J Med 2017;377(18):1713–1722;
- Arnold DW, et al. Muscle Nerve 2015;51(2):157–167.
- European Commission. Zynteglo. Available at: https://ec.europa.eu/health/documents/community-register/html/h1367.htm. Accessed October 4, 2019;
- Bluebird Bio. Press release. June 3, 2019. Available at: http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-eu-conditional-marketing-authorization. Accessed October 4, 2019;
- Zynteglo® [summary of product characteristics]. 2019. Available at: https://www.ema.europa.eu/en/documents/product-information/zynteglo-epar-product-information_en.pdf. Accessed October 4, 2019.
- Novartis. News release. May 19, 2020. Available at: https://www.globenewswire.com/news-release/2020/05/19/2035354/0/en/Novartis-receives-EC-approval-and-activates-Day-One-access-program-for-Zolgensma-the-only-gene-therapy-for-spinal-muscular-atrophy-SMA.html. Accessed June 1, 2020;
- Zolgensma® [summary of product characteristics]. 2020. Available at: https://www.ema.europa.eu/en/documents/product-information/zolgensma-epar-product-information_en.pdf. Accessed June 1, 2020.
- Amgen. News release. October 27, 2015. Available at: https://www.amgen.com/media/news-releases/2015/10/fda-approves-imlygic-talimogene-laherparepvec-as-first-oncolytic-viral-therapy-in-the-us/. Accessed June 1, 2020;
- Imlygic® [package insert]. 2019. Available at: https://www.pi.amgen.com/~/media/amgen/repositorysites/pi-amgen-com/imlygic/imlygic_pi.pdf. Accessed June 1, 2020;
- Amgen. News release. December 17, 2015. Available at: https://www.amgen.com/media/news-releases/2015/12/european-commission-approves-amgens-imlygic-talimogene-laherparepvec-as-first-oncolytic-immunotherapy-in-europe/. Accessed June 1, 2020;
- Imlygic™ [summary of product characteristics]. 2019. Available at: https://www.ema.europa.eu/en/documents/product-information/imlygic-epar-product-information_en.pdf. Accessed June 1, 2020.