NCT04925206

A Multicenter, Open Label Phase 1 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion Dependent Β-Thalassaemia

Study Summary

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

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Interventions

ET-01BIOLOGICAL

Recruited participants will receive ET-01 IV infusion following myeloablative conditioning with busulfan.

Study Locations

Facility	City	State	Country
Nanfang Hospital of Southern Medical University	Guangzhou	Guangdong	China
Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong	China
Shenzhen Children's Hospital	Shenzhen	Guangdong	China
Institute of Hematology & Blood Diseases Hospital	Tianjin	Tianjin Municipality	China

Official Trial Information

View on ClinicalTrials.gov

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