30 November 2023 - Previously reported results from ongoing proof of concept study showed extensive metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal health.

Orchard Therapeutics today announced the US FDA has granted fast track designation to OTL-203, an investigational haematopoietic stem cell gene therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I.

Read Orchard Therapeutics press release