Orchard Therapeutics develops autologous ex vivo gene therapies targeting rare diseases. Its portfolio includes Strimvelis, an EMA-approved therapy for ADA-SCID, and multiple clinical programs across primary immune deficiencies, neurometabolic disorders and hemoglobinopathies. The company has three advanced registrational studies for ADA-SCID, metachromatic leukodystrophy (MLD) and Wiskott–Aldrich syndrome (WAS), plus clinical programs for X-linked chronic granulomatous disease and transfusion-dependent beta-thalassemia and an extensive preclinical pipeline. Orchard says it will use new funding to advance its most advanced programs—OTL-101, OTL-200 and OTL-103—toward registration and commercialization. It also plans to support broader clinical and preclinical development across its rare disease gene therapy pipeline. The company operates from offices in the U.K. and the U.S., including London, San Francisco and Boston, and is led by president and CEO Mark Rothera and CFO/chief business officer Frank Thomas. Orchard Therapeutics is a drug developer advancing gene therapies to treat rare inherited disorders. The company is preparing to launch its gene therapy for a rare inherited disorder. It raised $110 million in a second round of funding to support these plans. Investors in the round included Baillie Gifford, ORI Capital, and Singapore's Temasek. Orchard said the proceeds will be used to launch the therapy and to beef up its manufacturing facilities. No further financial details were provided in the article. Orchard Therapeutics focuses on ex vivo autologous haematopoietic stem cell gene therapy, modifying a patient’s own stem cells with a functional gene copy before reintroduction. The company launched with operations in London and the U.S. and has formed formal partnerships with UCL, Great Ormond Street Hospital, the University of Manchester, UCLA, and Boston Children’s Hospital. Its lead development programs target primary immune deficiencies such as ADA-SCID, with interim data showing significant immune reconstitution and 100% survival in 32 patients treated at GOSH and UCLA as of March 2016. Orchard is also exploring lentiviral stem cell gene therapy for mucopolysaccharidosis type IIIA (MPS IIIA). Management includes senior pharma and gene therapy leaders, including founders Andrea Spezzi and Nicolas Koebel, and scientific leadership from Bobby Gaspar and other UCL/GOSH investigators. The company says it uses a collaborative development model to translate preclinical and early clinical research into commercial medicines.

Total raised

$291M

Funding rounds

3

Latest round

Series C

Latest activity

Aug 2018