Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

Summary

This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic stem cells in subjects with β-thalassemia major.

Timeline

Start

2023-12-25

Primary completion

2024-09-08

Completion

2024-11-30

Outcome

Outcome not reported

Stopped (Business): “Sponsor decision”

Drugs

Indications