Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
A biopharmaceutical company developing gene therapies for rare diseases.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Gene therapy shows promise for rare fatal 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 and severe brain disease. The treatment uses the child's own blood stem cells, modified to produce a missing enzyme, and aims to slow or stop disease progres…
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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Gene therapy offers hope for children with rare brain disease
Disease control CompletedThis study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare, inherited brain disease that causes severe disability and early death. The treatment uses the child's own blood stem cells, which are modified in a lab to carry a working copy of the mi…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC