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YOLT-204 in Pediatric Hemoglobinopathies
NCT07190001Phase 1Not yet recruiting
- Start
- Sep 2025
- Primary completion
- Apr 2027
- Modality
- Gene Editing, Gene Silencing, Lipid-based Nanoparticles
- Countries
- 1
01
Overview
Phase 1, open-label trial of YOLT-204 in beta thalassemia and sickle cell disease (SCD). Primary endpoints: Adverse event rate at 52 weeks, 3 months of sustained transfusion reduction at 52 weeks and 1 more. Planned start Sep 2025; status: not yet recruiting.
02
Drugs
YOLT-204
03
Conditions
beta thalassemiasickle cell disease (SCD)
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