Orchard Therapeutics is a leading global fully integrated commercial-stage biotech company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies.
Evolved from over 20 years of academic research, Orchard has developed a unique expertise in the manufacturing, preclinical and clinical development of gene therapies for rare diseases. To date, more than 130 patients have been treated with autologous ex vivo gene therapy across five different disease areas, with evidence of sustained clinical effects up to 17 years post treatment in some patients. The company's most advanced clinical program, OTL-101 for ADA-SCID (adenosine deaminase severe combined immunodeficiency), is expected to progress to a BLA (biological license application) with the FDA in 2018.
Orchard's portfolio of autologous ex vivo gene therapy programs include Strimvelis, the first autologous ex vivo gene therapy approved by the EMA in 2016, three programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (WiskottAldrich syndrome) and ADA-SCID (adenosine deaminase severe combined immunodeficiency), other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline.
The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles, Boston Children's Hospital, and Telethon Institute for Gene Therapy/Ospedale San Raffaele.
Orchard is privately held with offices in the UK and the US, including London, San Francisco and Boston. The company raised $110 million in a Series B in December 2017, was named a Fierce 15 Company by FierceBiotech in 2016 and was awarded a $19 million grant from the California Institute of Regenerative Medicine (CIRM).