Orchard Therapeutics plc - ADR

Orchard Therapeutics Plc is a biopharmaceutical company, which engages in discovering, acquiring, developing and commercializing gene therapies for patients with rare disorders. The company focuses on its autologous ex vivo gene therapy approach on three therapeutic rare disease franchise areas: primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies. Its portfolio includes Strimvelis, a commercial-stage gamma retroviral-based product for the treatment of Adenosine deaminase deficiency (ADA-SCID). The company was founded by Nicolas Koebel and Andrea Spezzi in August 2018 and is headquartered in London, the United Kingdom.

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Orchard Therapeutics is a fully integrated commercial-stage company dedicated to treating the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott-Aldrich syndrome), and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 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 and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele.

Orchard Therapeutics plc (ORTX) CEO Mark Rothera on Q2 2019 Results - Earnings Call Transcript

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Orchard Therapeutics PLC (ORTX) CEO Mark Rothera on Q4 2018 Results - Earnings Call Transcript

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