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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Own stem cells Gene-Hacked to fight rare hurler syndrome
Disease control OngoingThis study tests a one-time gene therapy for children with Hurler syndrome, a severe genetic disorder. Doctors take the child's own blood stem cells, add a working copy of the missing gene, and put them back. The goal is to help the body produce the enzyme it lacks, potentially s…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Gene therapy could replace risky transplants for kids with hurler syndrome
Disease control OngoingThis Phase 3 trial tests a new gene therapy called OTL-203 against the standard stem cell transplant for children with Hurler syndrome, a rare genetic disorder. The therapy uses the child's own blood stem cells, modified to produce the missing enzyme, aiming to improve survival a…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC
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Gene therapy hope for rare brain disease in children
Disease control OngoingThis study tests a single infusion of OTL-200 gene therapy in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The therapy uses the patient's own blood stem cells, modified to produce a missing enzyme. Resea…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 13:33 UTC