shares of Orchard Therapeutics PLC (ORTX) on
Orchard Therapeutics Plc is a biopharmaceutical company, which engages in discovering, acquiring, developing and commercializing gene therapies for patients with rare disorders. The company 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 firm 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. The company 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.