Orchard Therapeutics plc develops gene therapies for rare diseases with a single administration approach, transforming a patient's stem cells into gene-modified cellular drug products. It provides Strimvelis, clinical development products, and preclinical programs for the treatment of diseases like ADA-SCID, metachromatic leukodystrophy, and mucopolysaccharidosis. The company also has a collaboration agreement with Pharming Group N.V. for the treatment of hereditary angioedema.