FDA supports clinical trials for the definitive cure of beta thalassaemia through genetic engineering

The FDA supports two potential definitive treatments for beta thalassaemia that stem from the companies Bluebird bio and Sangamo BioSciences

March 4, 2015

State of the art scientific researches to achieve the cure for beta thalassaemia are closer than ever to reach their goals. The U.S. Food and Drug Administration (FDA) supports two potential definitive treatments for beta thalassaemia that stem from the companies Bluebird bio and Sangamo BioSciences.

Bluebird bio, a clinical-stage company that aims to develop gene therapies for severe genetic and rare diseases, has been granted by the FDA with the Breakthrough Therapy designation to its LentiGlobin® BB305 Drug Product for the treatment of transfusion-dependent patients with beta-thalassaemia major. The lentiviral-based gene therapy consists of inserting a functional beta-globin gene into patients’ hematopoietic stem progenitor cells (HSPCs) ex vivo and reinfusing them into the bloodstream once the cells are modified (autologous stem cell transplantation). LentiGlobin® BB305 Drug Product is currently used in two Phase 1/2 clinical trials carried out in United States, Australia, Thailand and France.

On the other hand, Sangamo BioSciences, in collaboration with Biogen Idec, has developed a genome editing approach through Zinc Finger Nucleases (ZFN) technology that allows the gene correction of HSPCs as a potential one-time lasting therapy for beta thalassaemia. "We are using our genome editing technology to target a key genetic switch in a patient's own HSPCs to enable continued production of fetal hemoglobin in the red blood cells of adults. We know that elevated production of fetal globin can ameliorate disease symptoms of haemoglobinopathies such as beta-thalassaemia. We are also developing this strategy for sickle cell disease." stated Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. The FDA acceptance of Sangamo’s Investigational New Drug enables to initiate a Phase 1/2 clinical trial of the ZFP Therapeutic in transfusion-dependent patients with beta-thalassaemia major.

Bone marrow transplant of HSPCs from a compatible donor is the only curative treatment so far, however, this therapy is limited due to the difficulty of finding matched donors and the risk of graft versus host disease after transplantation of the foreign cells. By performing these groundbreaking approaches, both disadvantages are prevented.

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