Bluebird Bio has won $9.3m award from the California Institute for Regenerative Medicine (CIRM) to advance the gene therapy clinical development.
The CIRM award, the first under the agency’s Strategic Partnership Awards initiative, is intended to support a Phase I/II study designed to assess the safety and efficacy of LentiGlobin.
Trials of LentiGlobin, the company’s development-stage programme for the treatment of beta-thalassemia, are scheduled to begin in 2013 in the US.
Bluebird Bio chief medical officer Dr David Davidson said the clinical data generated to date demonstrated the potential of LentiGlobin as a one-time transformative gene therapy for patients with beta-thalassemia.
"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 clinical indications," Davidson said.
"We are delighted that CIRM has chosen to recognise the importance of this innovative approach for the treatment of one of the most commonly inherited blood disorders, and we are excited to work with CIRM to continue the development of LentiGlobin in the U.S."
LentiGlobin delivers a fully functional human beta-globin gene into the patient’s own hematopoietic stem cells to produce fully functioning red blood cells.
The company is presently conducting a Phase I/II trial evaluating LentiGlobin in the treatment of beta-thalassemia and sickle cell disease.
Results of the first patient demonstrated stable expression of functional beta-globin resulting in transfusion independence, which at present extends for more than four years subsequent to a single treatment, according to the company.