Orchard Therapeutics
Clinical trials sponsored by Orchard Therapeutics, explained in plain language.
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One-Time treatment aims to halt devastating childhood brain disease
⭐️ CURE ⭐️ CompletedThis study tested a one-time gene therapy called OTL-200 for children with early-onset metachromatic leukodystrophy (MLD), a rare and fatal genetic brain disease. The treatment involved collecting a child's own blood stem cells, fixing the genetic error in a lab, and returning th…
Phase: PHASE2 • Sponsor: Orchard Therapeutics • Aim: ⭐️ CURE ⭐️
Last updated Mar 18, 2026 14:41 UTC
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Doctors test gene fix for devastating childhood brain disease
Disease control CompletedThis early-stage trial tested a gene therapy for metachromatic leukodystrophy (MLD), a rare and severe genetic disorder that damages the brain and nervous system. Researchers collected blood stem cells from 20 pre-symptomatic or early-symptomatic children with MLD, used a modifie…
Phase: PHASE1, PHASE2 • Sponsor: Orchard Therapeutics • Aim: Disease control
Last updated Mar 09, 2026 14:23 UTC