Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
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One-Time gene infusion offers hope for rare brain disease
Disease control OngoingThis study tests a one-time gene therapy called OTL-200 in 6 people with late juvenile metachromatic leukodystrophy (MLD), a rare genetic disease that damages the brain and nerves. The treatment uses the patient's own blood stem cells, modified to produce a missing enzyme, and ai…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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Experimental gene therapy aims to fix rare childhood disease
Disease control OngoingThis study tests a new gene therapy for children with MPS I Hurler, a rare genetic disease that damages organs. The treatment takes the child's own blood stem cells, adds a working copy of the missing gene, and puts them back. The goal is to help the body produce the missing enzy…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 14, 2026 12:04 UTC
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Gene therapy may offer safer option for rare childhood disease
Disease control OngoingThis study tests a new gene therapy called OTL-203 for children with Hurler syndrome, a rare genetic disorder that damages organs and the brain. It compares the gene therapy to the current standard treatment, a stem cell transplant. The goal is to see if OTL-203 can improve survi…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 12, 2026 13:40 UTC