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OTL-200 in Late Juvenile Metachromatic Leukodystrophy
2019-002636-82Phase 3Active
- Sponsor
- Orchard Therapeutics
- Start
- Jul 2019
- Modality
- Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of atidarsagene autotemcel in metachromatic leukodystrophy (MLD), sponsored by Orchard Therapeutics. Primary endpoints: Pharmacodynamic effect in CSF and brain and Pharmacodynamic effect in bone marrow and peripheral blood. Started Jul 2019; status: active.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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