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OTL-200 cryopreserved autologous CD34+ gene therapy for early-onset metachromatic leukodystrophy
2017-001730-26Phase 3Active
- Sponsor
- Orchard Therapeutics
- Start
- Apr 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 endpoint: GMFM score at 24 months post gene-therapy. Started Apr 2019; status: active.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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