Bluebird Bio has moved a step closer to receiving European approval for the first gene therapy designed to treat beta-thalassemia. The company's treatment, Lentiglobin, has been recommended for approval by the EU's Committee for Medicinal Products for Human Use, the drug-reviewing arm of the European Medicines Agency. Lentiglobin works by using an inactivated virus to insert a healthy copy of the beta-globin gene into a patient’s bone marrow, producing red blood cells and reducing the need for transfusions. In its application submitted in July, Bluebird said it had completed clinical trials on mice that showed significant improvement after just one dose of the treatment.
Bluebird Bio has moved a step closer to receiving European approval for the first gene therapy designed to treat beta-thalassemia. The company's treatment, Lentiglobin, has been recommended for approval by the EU's Committee for Medicinal Products for Human Use, the drug-reviewing arm of the European Medicines Agency. Lentiglobin works by using an inactivated virus to insert a healthy copy of the beta-globin gene into a patient’s bone marrow, producing red blood cells and reducing the need for transfusions. A final decision on whether to approve the treatment is expected within the next three months.