newsfilter
← Latest News
GlobeNewswire·

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient's own hematopoietic stem cells HURCULES study to