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BRL-101 in Severe Sickle Cell Disease
NCT06300723Phase 2Enrolling by invitation
- Sponsor
- BRL Medicine Inc.
- Start
- Jul 2024
- Primary completion
- Mar 2026
- Modality
- Gene Silencing, CRISPR-Cas9, Hematopoietic Stem Cells
- Countries
- 1
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. Started Jul 2024; status: enrolling by invitation.
02
Drugs
BRL-101
03
Conditions
sickle cell disease (SCD)
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