Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia

Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia

Ex vivo autologous hematopoietic stem cell lentiviral-based gene therapy with betibeglogene autotemcel has been studied in patients with transfusion-dependent β-thalassemia in Phase III clinical trials (HGB-207 and HGB-212), with 90% of patients reaching transfusion independence (TI). Here, we explo...

Full description

Bibliographic Details
Main Authors: Dustin Whitney, Ilya Shestopalov, Maeva Fincker, Marc d’Anjou, Kelly Kral, Marisa Gayron, Francis J. Pierciey, Richard A. Colvin
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
lentiviral-based gene therapy
transfusion-dependent thalassemia
multivariate analysis
transduction efficiency
drug product quality attributes
Zynteglo
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001948

Internet

http://www.sciencedirect.com/science/article/pii/S2329050123001948

Similar Items