Gene Therapy for Transfusion Dependent Beta-thalassemia

计划入组 10 人 · 首次发布于 2015 年 5 月 25 日

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

计划入组 12 人 · 首次发布于 2024 年 4 月 15 日

Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.

计划入组 15 人 · 首次发布于 2026 年 2 月 25 日

vebeglogene autotemcel

γ-globin gene therapy(CSL Behring LLC)

beta-globin restored autologous hematopoietic stem cell therapy (beta-thalassemia major)

Beta Bionics

Beta Innov

France

K-Group Beta, Inc.

United States

EMA Approves Bluebird Bio's Zynteglo, First Gene Therapy for Beta Thalassemia

YolTech Therapeutics Launches Clinical Trial for In Vivo Gene Editing Therapy YOLT-204 in Transfusion-Dependent Beta-Thalassemia

Kanglin Biotechnology Secures $20 Million to Advance KL003 Gene Therapy for Beta Thalassemia and Sickle Cell Disease