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Gene Therapy for Metachromatic Leukodystrophy (Phase I/II)
2024-515253-25-00Phase 2Completed
- Sponsor
- Orchard Therapeutics
- Start
- Oct 2024
- Modality
- Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of atidarsagene autotemcel in metachromatic leukodystrophy (MLD), sponsored by Orchard Therapeutics. Primary endpoints: Conditioning regimen-related safety, Short-term safety and tolerability of LV-transduced cell infusion and 3 more. Started Oct 2024; status: completed.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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Competitive context
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