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OTL-200 Gene Therapy in Early Onset Metachromatic Leukodystrophy
2024-511970-66-00Phase 3Completed
- Sponsor
- Orchard Therapeutics
- Start
- Dec 2017
- 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 endpoint: Gross motor function measure (GMFM) score at 24 months post gene therapy. Started Dec 2017; status: completed.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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