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OTL-103 Gene Therapy in Wiskott-Aldrich Syndrome
2018-003842-18Phase 3Active
- Sponsor
- Orchard Therapeutics
- Start
- Nov 2018
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of etuvetidigene autotemcel in Wiskott-Aldrich syndrome (WAS) and Immunogenetic disorder, sponsored by Orchard Therapeutics. Primary endpoint: Engraftment and biological efficacy at 6 months. Started Nov 2018; status: active.
02
Drugs
etuvetidigene autotemcel
03
Conditions
Wiskott-Aldrich syndrome (WAS)Immunogenetic disorder
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