Advertisement

LentiGlobin gene therapy for transfusion-dependent β-Thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study

      Background: Gene therapy could provide a transformative treatment for patients with transfusion-dependent β-thalassemia (TDT). HGB-204 is a multi-center Phase 1/2 clinical study investigating the safety and efficacy of LentiGlobin BB305 Drug Product (DP) in patients with TDT. We report 3.7–27.0 months follow up on the study's full cohort.
      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Cytotherapy
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect