Orchard Therapeutics

Orchard Therapeutics is a gene therapy company developing ex vivo haematopoietic stem cell therapies for rare genetic diseases, primarily severe neurometabolic and immunological disorders in children.

Its platform modifies a patient's own stem cells with a functional copy of the defective gene before reinfusion.

Lead product Libmeldy (atidarsagene autotemcel) is approved in the UK and EU for early-onset metachromatic leukodystrophy.

Further programmes target mucopolysaccharidosis disorders. The company was acquired by Kyowa Kirin in January 2024.