Our Story
We’re part of gene therapy history — and its future.
Orchard was founded in 2015—but our roots run deeper, going back to some of the first research and clinical development involving hematopoietic stem cell, or HSC, gene therapy. Our team has played a central role in the evolution of this technology from a promising idea to a potentially life-transforming reality. Today, we carry forward our history of visionary science to imagine new possibilities for people and families affected by genetic and other severe diseases.
Explore some of the history of HSC gene therapy below and imagine where we could go next.
ex vivo autologous gene therapy history
Orchard company history
Orchard program history
1990
The first clinical trial of an ex vivo autologous gene therapy begins at the National Institutes of Health. In the trial, two children with adenosine deaminase severe combined immunodeficiency (ADA-SCID) receive regular infusions of a gene-corrected version of their own cells. Source: Blaese RM et al. Science. 1995;270(5235):475–480.1993
Two of Orchard’s scientific founders, Bobby Gaspar, M.D., Ph.D., and Donald Kohn, M.D., are involved in some of the first clinical studies of an ex vivo autologous gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID).Sources: Kohn DB et al. Nat Med. 1998 Jul;4(7):775-80.; Hoogerbrugge PM et al. Gene Ther. 1996 Feb;3(2):179-83.1995
Results from the first clinical trial of an ex vivo autologous gene therapy are published. The data show that ex vivo autologous gene therapy may have the potential to be a therapeutic option for certain patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID).Source: Blaese RM et al. Science. 1995;270(5235):475–480.2000
Researchers in France report successful results of a clinical trial of an ex vivo autologous gene therapy. In the trial, the therapy provided full correction of disease phenotype in three infants with a form of severe combined immunodeficiency.Source: Cavazzana-Calvo M et al. Science. 2000;288(5466):669–672.2001
Two of Orchard’s scientific founders, Bobby Gaspar, M.D., Ph.D., and Adrian Thrasher, M.D., Ph.D., treat the first child in the UK with an ex vivo autologous gene therapy. The therapy is intended to treat a form of severe combined immunodeficiency.Source: Gaspar HB et al. Lancet. 2004 Dec 18-31;364(9452):2181-7.October 2010
GlaxoSmithKline (GSK) announces a research collaboration with the San Raffaele Telethon Institute for Gene Therapy, with the goal of advancing the development of certain ex vivo autologous gene therapies. The collaboration gives rise to clinical-stage programs for adenosine deaminase severe combined immunodeficiency (ADA-SCID), metachromatic leukodystrophy (MLD), Wiskott Aldrich syndrome (WAS) and beta thalassemia that are later acquired by Orchard.2012
The first gene therapy for an inherited disease is approved by the European Medicines Agency (EMA).Source: “European Medicines Agency recommends first gene therapy for approval.” https://www.ema.europa.eu/en/news/european-medicines-agency-recommends-first-gene-therapy-approval (accessed Oct. 7, 2019)Note: Not an Orchard product.2012
Two of Orchard’s scientific founders, Bobby Gaspar, M.D., Ph.D., and Adrian Thrasher, M.D., Ph.D., treat the first patient in a clinical trial at Great Ormond Street Hospital in London (later a collaboration partner of Orchard’s) with an ex vivo autologous gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID).Source: Gaspar HB et al. Molecular Therapy. 23: S102-S103. 01 May 2015.2013
Two of Orchard’s scientific founders, Bobby Gaspar, M.D., Ph.D., and Adrian Thrasher, M.D., Ph.D., treat the first patient in a clinical trial at Great Ormond Street Hospital in London (later a collaboration partner of Orchard’s) with an ex vivo autologous gene therapy for X-linked chronic granulomatous disease (X-CGD).Source: Malik MA, Masab M. Wiskott-Aldrich Syndrome. [Updated 2019 Jun 22]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2019 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK539838/September 2015
Orchard Therapeutics is founded.January 2016
Orchard opens its first office in London. The company later moves into larger office space as it grows.April 2016
Strimvelis® becomes the first ex vivo autologous gene therapy approved by the European Medicines Agency (EMA). Orchard later acquires Strimvelis. Strimvelis is not approved outside of the European Union.Strimvelis (autologous CD34+ cells transduced to express ADA) has been approved by the European Medicines Agency. It has not been approved by the U.S. Food and Drug Administration. For more information about Strimvelis, please see the EU Summary of Product Characteristics available on the EMA website.May 2016
Orchard launches with a $33 million Series A financing led by F-Prime Capital. At the time of launch, the company has development programs in adenosine deaminase severe combined immunodeficiency (ADA-SCID) and mucopolysaccharidosis type IIIA (MPS-IIIA).October 2016
Orchard establishes its U.S. operations with the opening of an office in Foster City, California. The company later moves its California operations to Menlo Park.August 2017
An ex vivo autologous gene therapy is approved by the U.S. Food and Drug Administration (FDA) for the first time.Source: “FDA approval brings first gene therapy to the United States.” https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states (accessed Oct. 18, 2019)Note: Not an Orchard productNovember 2017
Orchard licenses a preclinical program in mucopolysaccharidosis type IIIB (MPS-IIIB) from The University of Manchester, UK.December 2017
Orchard announces a strategic alliance with Généthon to develop an ex vivo autologous gene therapy for X-linked chronic granulomatous disease (X-CGD).December 2017
For the first time, the U.S. Food and Drug Administration (FDA) approves a gene therapy for an inherited disease.Source: “FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss.” https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss (accessed Oct. 7, 2019)April 2018
Orchard acquires GlaxoSmithKline’s portfolio of gene therapies for rare inherited diseases, including Strimvelis® and clinical-stage programs for metachromatic leukodystrophy (MLD), Wiskott Aldrich syndrome (WAS) and beta thalassemia.Strimvelis® has been approved by the European Medicines Agency. It has not been approved by the U.S. Food and Drug Administration. The other therapies in our pipeline are currently in clinical trials and have not been approved by any regulatory agency or health authority. For more information about Strimvelis, visit the EMA website.April 2018
Orchard opens a new U.S. office in Boston’s Seaport District.October 2018
Orchard lists on the U.S. Nasdaq stock exchange under the ticker symbol “ORTX.”May 2019
Orchard licenses a clinical-stage program in mucopolysaccharidosis type I (MPS-I) from the San Raffaele Telethon Institute for Gene Therapy.December 2020
Libmeldy™ becomes Orchard’s second ex vivo autologous gene therapy approved by the European Medicines Agency (EMA). Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway.Libmeldy (atidarsagene autotemcel), also known as OTL-200,has been approved by the European Comission. OTL-200, is an investigational therapy in the US. For more information about Libmeldy, please see the EU Summary of Product Characteristics available on the EMA website.
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