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OTL-201 Gene Therapy for MPS IIIA
NCT04201405Phase 2Active, not recruiting
- Sponsor
- The University of Manchester
- Start
- Jan 2020
- Primary completion
- Oct 2024
- Modality
- Gene Editing, Gene Silencing, Hematopoietic Stem Cells
- Countries
- 1
01
Overview
A Phase 2 trial of OTL-201 in mucopolysaccharidosis type III A, sponsored by The University of Manchester. Started Jan 2020; status: active, not recruiting.
02
Drugs
OTL-201
03
Conditions
mucopolysaccharidosis type III A
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