A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with -Thalassemia Major by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral A-T87Q-Globin Vector (

Project: Research project

Project Details

StatusFinished
Effective start/end date10/16/1310/31/19

Funding

  • bluebird bio, Inc. (HGB-204)