Orchard Therapeutics

This study is testing whether a new gene therapy called OTL-203 works better and is safer than the current standard treatment (stem cell transplant) for children with Hurler syndrome. Hurler syndrome is a severe genetic disorder that causes progressive damage to the brain, bones,…

This study is testing a single infusion of an experimental gene therapy called OTL-200 for children and teenagers with a rare, progressive brain disease called Late Juvenile Metachromatic Leukodystrophy (MLD). The therapy involves collecting a patient's own blood stem cells, addi…

This study is testing a one-time gene therapy for children with Hurler syndrome, a severe and life-limiting genetic disorder. Doctors collect a child's own blood stem cells, add a corrected gene in the lab, and then return the cells to the child. The main goals are to see if this…