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 in up to 6 children with late juvenile metachromatic leukodystrophy (MLD), a rare and severe genetic brain disease. The therapy aims to correct the underlying genetic cause by giving the patien…

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…