ORTX

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 cellular 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-203 for mucopolysaccharidosis type I, OTL-201 for mucopolysaccharidosis type MPS-IIIA, and OTL-202 for mucopolysaccharidosis type IIIB. It has a strategic collaboration agreement with Pharming Group N.V. for research, development, manufacturing, and commercialization of OTL-105, an investigational ex vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom.