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Safety and Efficacy of BRL-101 in Sickle Cell Disease
NCT06287099Phase 2Not yet recruiting
- Sponsor
- BRL Medicine Inc.
- Start
- Apr 2024
- Primary completion
- Oct 2025
- Modality
- Gene Silencing, CRISPR-Cas9, Hematopoietic Stem Cells
01
Overview
Phase 2, open-label, single-arm trial of BRL-101 in sickle cell disease (SCD), sponsored by BRL Medicine Inc. Primary endpoints: Proportion of stem cell engrafted subjects at 42 days, Time to neutrophil engraftment at 42 days and 1 more. Planned start Apr 2024; status: not yet recruiting.
02
Drugs
BRL-101
03
Conditions
sickle cell disease (SCD)
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