Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

Summary

This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Timeline

Start

2017-12-01

Primary completion

2019-01-01

Completion

2020-12-31

Drugs

Indications