Orchard Therapeutics plc, a biopharmaceutical company, develops gene therapies for serious and life-threatening rare diseases in the United Kingdom, European Union, and the United States. 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. It provides 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 MPS-IIIA and OTL-202 for mucopolysaccharidosis type IIIB. Orchard Therapeutics plc entered into a manufacturing and technology development agreement with MolMed S.p.A. to develop, manufacture and supply certain viral vectors and conduct cell processing activities for certain Orchard development and commercial programs. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is based in London, the United Kingdom.

Healthcare

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