Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Summary

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with transfusion-dependent β-thalassemia.

Timeline

Start

2024-01-04

Primary completion

2025-12-31

Completion

2026-12-31

Drugs

Indications