Orchard Therapeutics (ORTX)

Orchard Therapeutics plc is a gene therapy company that researches, develops, and commercializes hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand, and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises a clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA. Additionally, there is a pre-clinical program, OTL-204, for the treatment of frontotemporal dementia with progranulin mutations. In addition, Orchard Therapeutics develops programs for immunological disorders, which consist of pre-clinical programs, including OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2, and OTL-105 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. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd.