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

Official 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).

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 3 participants

Interventions

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

Primary Outcomes

• Proportion of stem cell engrafted subjects (Within 42 Days After BRL-101 Infusion)
• Time to neutrophil engraftment (Within 42 Days After BRL-101 Infusion)
• Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion (Up to 12 Months After BRL-101 Infusion)

Trial Locations

• First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China

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