Key clinical point: Gene therapy engraftment was achieved sooner using intrabone delivery.
Major finding: After a median of 16 months of follow-up, five of seven patients who received this novel gene therapy needed markedly fewer blood transfusions than at baseline.
Data source: An interventional phase 1/2 trial of seven patients with transfusion-dependent beta thalassemia.
Disclosures: Telethon Foundation provided funding. Dr. Marktel disclosed research funding from GlaxoSmithKline, which has licensed the therapy.
Source: Marktel S et al. ASH 2017 Abstract 355.
Marktel S et al. ASH 2017 Abstract 355
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