Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
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Stem cell gene therapy offers hope for children with fatal brain disease
Disease control CompletedThis study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and fatal genetic disease that damages the brain and nerves. Doctors took stem cells from each child, added a working copy of the faulty gene, and put the cells back after mild chemother…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 12:06 UTC
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Gene therapy shows promise for rare, devastating childhood disease
Disease control CompletedThis study tested a one-time gene therapy (OTL-200) in 10 children with early-onset metachromatic leukodystrophy (MLD), a severe genetic disorder that damages the nervous system. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme. The main g…
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 19, 2026 12:00 UTC