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Clinical trial updates


An agent that regulates hemoglobin gene expression, used for the treatment of other haematological conditions, such as myelofibrosis and resistant to hydroxyurea polycythemia vera. A Phase 2a trial (TRUTH) in 30 transfusion-dependent β-thalassemia with splenomegaly showed that ruxolitinib therapy for 30 weeks resulted in slight improvement in pre-transfusion haemoglobin levels ...

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LentiGlobin gene therapy

Autologous CD34+ cells transduced with the BB305 lentiviral vector. An initial phase 1/2 study [Northstar (HGB-204)] in 22 β-thalassaemia patients showed transfusion-independency for non-β0/β0 genotypes and 60% reduction in transfuions in β0/β0 genotype. The results were published in N Engl J Med, in April 19, 2018. An ongoing phase 2 study (HGB-205) evaluates ...

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