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Clinical Trial 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).


Clinical Trial Description

This clinical trial is a single-arm, single-dose, single center, open-label study without dose escalation. The primary objective is to explore the safety of the study drug in SCD. Myeloablative conditioning and administration for the remaining subjects can only be started after the first subject completes dosing and safety observation and assessment. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT06287099
Study type Interventional
Source Bioray Laboratories
Contact Xiaoqin Feng, PhD
Phone 020-61641921
Email fxq126126@126.com
Status Not yet recruiting
Phase N/A
Start date April 20, 2024
Completion date May 10, 2026

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