Orchard Therapeutics develops ex vivo autologous HSC gene therapies for rare genetic diseases; its Libmeldy is the first FDA-approved therapy for metachromatic leukodystrophy.
Orchard Therapeutics is a gene therapy company developing ex vivo autologous hematopoietic stem cell (HSC) gene therapies for rare genetic and other severe diseases. The approach involves collecting a patient's own blood stem cells, modifying them outside the body to correct the underlying genetic cause of disease, and reinserting them as a single treatment. The company was founded in 2015 and operates in the United States and the United Kingdom.
Its scientific roots trace to 2001, when founders Dr. Bobby Gaspar and Dr. Adrian Thrasher treated the first child with an ex vivo autologous gene therapy in the UK. In 2018 Orchard Therapeutics acquired GlaxoSmithKline's gene therapy portfolio. Its lead product, Libmeldy®, is an ex vivo autologous HSC gene therapy for children with metachromatic leukodystrophy (MLD) and became the first and only FDA-approved therapy in the U.S. for MLD in 2024.
The company's technical work spans hematopoietic stem cell gene therapy, cell and gene therapy, and therapeutics for rare genetic disease, with applications in pediatric medicine. Orchard Therapeutics is a wholly owned subsidiary of Kyowa Kirin and has a global presence.
