Orchard Therapeutics Limited

Orchard Therapeutics plc, an integrated biopharmaceutical company, focuses on developing various transformative treatments. It dedicated to transform the lives of patients with serious and life-threatening rare diseases through autologous ex vivo gene therapies. The company's gene therapy approach seeks to transform a patient's hematopoietic stem cells into a gene-modified drug product to treat the patient's disease through a single administration. Its commercial stage products include Strimvelis, a gammaretroviral-based product for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company's clinical development products comprise OTL-101 for the treatment of ADA-SCID; OTL-200 to treat metachromatic leukodystrophy; OTL-103 for the treatment of Wiskott-Aldrich syndrome; OTL-102 for X-linked chronic granulomatous disease; and OTL-300 for transfusion-dependent beta-thalassemia. Its preclinical programs include OTL-201 for mucopolysaccharidosis type IIIA and OTL-202 for mucopolysaccharidosis type IIIB. The company, formerly known as Orchard Rx Limited, was founded in 2015 and is headquartered in London, the United Kingdom.