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Phase II open-label single-arm study of CS-101 in transfusion-dependent severe beta-thalassemia
CTR20262399Phase 2Active
- Sponsor
- CorrectSequence Therapeutics
- Start
- Jun 2026
- Modality
- Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Cytosine Base Editors → Base Editing → Gene Editing → Gene Therapies, Adenine Base Editors → Base Editing → Gene Editing → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of CS-101 in beta thalassemia, sponsored by CorrectSequence Therapeutics. Primary endpoints: Proportion of participants achieving TI12 with weighted average hemoglobin at least 90 g/L, Adverse events and serious adverse events and 4 more. Started Jun 2026; status: active.
02
Drugs
CS-101
03
Conditions
beta thalassemia
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