Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
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Gene therapy breakthrough for rare childhood disease?
Disease control OngoingThis study tests a new gene therapy, OTL-203, for children with Hurler syndrome, a rare genetic disease 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 the gene therapy can prevent de…
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 12:12 UTC
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Gene therapy hope for kids with rare nerve 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 nervous system. The treatment uses the patient's own blood stem cells, modified to produce a missing enzyme, to slow …
Phase: PHASE3 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated May 26, 2026 12:02 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