Recently have been published the results of two separate clinical trials of two years each showing the potential of gene therapy with LentiGlobina to eliminate or reduce chronic blood transfusions in patients with β-thalassemia transfusion-dependent. Both studies, Northstar (HGB-204), recently completed, and HGB-205, currently underway, are aimed at evaluating the safety and efficacy of a single-dose treatment of gene therapy based on LentiGlobin and whose interim results showed that most of the 22 patients in the two Phase 1/2 trials followed for two or more years remained transfusion-free. From a safety point of view, the collected profile was consistent with myeloablative conditioning and the busulfan chemotherapeutic agent. The recently completed Northstar study is a multicentre, open-dose, single-dose, non-randomized, Phase 1/2 study designed to assess the safety and efficacy of LentiGlobin in the treatment of patients with TDT. HGB-205 is an ongoing, single-dose, non-randomized, monocentric, single-dose, open-label, 1/2 study designed to assess the safety and efficacy of LentiGlobin in the treatment of patients with TDT and severe sickle cell anemia (SCD). The HGB-205 Phase 1/2 study is conducted in a single center in France. It is designed to evaluate the safety and efficacy of LentiGlobin in the treatment of subjects with TDT and SCD. Seven subjects were enrolled in the study. The intention of the manufacturer is to present, at the end of this year, an authorization request for the LentiGlobina for β-thalassemia transfusion-dependent to the EU.

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