Clinical Study of BRL-101 in Severe SCD

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).

Timeline

Start

2024-07-29

Primary completion

2026-03-20

Completion

2026-06-15

Drugs

Indications