Gene therapy firm bluebird bio won a $9.3 million grant from the California Institute for Regenerative Medicine to support a Phase I/II study evaluating bluebird’s LentiGlobin® treatment for β-thalassemia. The trial is scheduled to start in the U.S. next year. The CIRM grant is one of the first to have been made through the Institute’s new Strategic Partnership Awards initiative.
bluebird is leveraging its gene therapy platform to develop treatments for severe genetic disorders. The technology is based on introducing the corrected version of a disease-causing gene into bone marrow stem cells taken from the patient, and then reintroducing these engineered cells as therapy. The firm’s lead Phase III-stage program is an ABCD-1 gene therapy for treating childhood cerebral adrenoleukodystrophy (CCALD), an inherited neurological disorder that in its most severe form causes damage to the myelin sheath and progressive dysfunction of the adrenal glands. bluebird raised $60 million in a series D financing round in July to support its clinical development programs.
The LentiGlobin candidate uses a patients’ own hematopoietic stem cells engineered to carry a fully functional human β-globin gene, which when transplanted back into the patient produce fully functioning red blood cells. Phase I/II trials of the product in the treatment of β-thalassemia and sickle cell disease are ongoing. Promising initial data were reported back in 2010, and showed that a single LentiGlobin treatment results in stable expression of functional β-globin, which has persisted for more than four years (results to date), and removes the need for blood transfusions.
“We are very encouraged by the clinical data generated to date demonstrating the potential of LentiGlobin as a one-time transformative gene therapy for patients with beta-thalassemia,” comments David Davidson, M.D., bluebird CMO. “Bluebird bio has made significant advances in lentiviral vector design, transduction efficiency, and, in our manufacturing process, enabling the production of gene-modified products that can be scaled and deployed for many different indications.”
ViaCyte has also today announced the award of a $10.1 million Strategic Partnership Award from CIRM to support IND filing and initial clinical evaluation of an implantable cell therapy for treating diabetes.