Orchard Therapeutics plc, formerly Orchard Rx Ltd, is a commercial-stage, fully-integrated biopharmaceutical company. It is engaged in manufacturing and commercialization of gene and cell therapies, position to provide transformative therapies to patients suffering from a range of rare diseases. The Company is focused on its autologous ex vivo gene therapy approach on three therapeutic rare disease franchise areas: primary immune deficiencies, neurometabolic disorders and hemoglobinopathies. Its portfolio includes Strimvelis, its commercial-stage gammaretroviral-based product for the treatment of ADA-SCID five lentiviral product candidates in clinical-stage development and several other product candidates in preclinical development. It is developing OTL-101 as an autologous ex vivo lentiviral gene therapy to sustainably treat patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID) through a single administration.