ORTX

Orchard Therapeutics plc, a biopharmaceutical company, develops gene therapies for serious and life-threatening rare diseases in the United Kingdom, European Union, and the United States. The company's gene therapy approach seeks 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. It provides Strimvelis, a gammaretroviral-based product for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID).