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NCT06300723

Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of CRISPR/Cas9-edited Autologous CD34 + Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Severe Sickle Cell Disease

Study Summary

This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101).

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Interventions

BRL-101DRUG
CD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.

Study Locations

FacilityCityStateCountry
First Affiliated Hospital of Guangxi Medical UniversityNanningGuangxiChina

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026