Home » Publications » Clinical trial updates

Clinical trial updates

Ruxolitinib

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 ...

Read More »

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 ...

Read More »