Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
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Gene therapy shows promise for rare, devastating brain disease in kids
Disease control CompletedThis study tested a gene therapy called OTL-200 in 10 children with early-onset metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme, and aims to slo…
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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Gene therapy offers hope for children with rare 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. The treatment involves taking the child's own blood stem cells, adding a working copy of the faulty gene, and giving the cell…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:55 UTC