Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Orchard Therapeutics Licenses Potentially Life-Changing MPS-I Gene Therapy Program
ADA Severe Combined Immune Deficiency (ADA-SCID), Business, Gene Therapy, Gene Transfer Technologies, Hematopoietic Stem Cell Transplantation (HSCT), Hurler syndrome (MPS I), Licensing, Neurometabolic Disorders, Stem Cell TherapyOrchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.
