Orchard Therapeutics plc

Orchard Therapeutics plc is a biopharmaceutical company that develops gene therapies for serious and life-threatening rare diseases. The company is headquartered in London, the United Kingdom, but currently operates in the United Kingdom, European Union, and the United States. Orchard Therapeutics was founded in 2015, and aims 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.

The company’s leading product is Strimvelis, a gammaretroviral-based therapy for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID); Orchard is also developing OTL-101 for the treatment of ADA-SCID and OTL-200 to treat metachromatic leukodystrophy. They also have 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.

In addition, Orchard is developing a number of preclinical programs, including OTL-203 for mucopolysaccharidosis type I, OTL-201 for mucopolysaccharidosis type MPS-IIIA, and OTL-202 for mucopolysaccharidosis type IIIB. They have 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.

Orchard Therapeutics plc currently has more than 230 employees, and aims to develop gene therapies that have the potential to drastically improve the lives of patients with rare diseases. The company has a strong focus on childhood diseases, and has a number of research collaborations with universities and academic institutions in the UK, Europe and the US. They are committed to continuing this research and development in order to offer effective treatments to as many patients as possible.