CAMBRIDGE, Mass.--(bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that the California Institute for Regenerative Medicine (CIRM) has approved an award to the company for $9.3 million for the first round of its new Strategic Partnership Awards initiative. The award is to support a Phase 1/2 study to evaluate the safety and efficacy of LentiGlobin®, the company’s development-stage program for the treatment of beta-thalassemia, which will be initiated in the United States in 2013.)--
“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”
“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,” said David Davidson, M.D., chief medical officer, bluebird bio. “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. We are delighted that CIRM has chosen to recognize 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.”
bluebird bio’s LentiGlobin product introduces a fully functional human beta-globin gene into the patient's own hematopoietic stem cells. These corrected stem cells ultimately produce fully functioning red blood cells. bluebird bio is currently conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell disease. Results of the first patient were reported in Nature in 2010, showing dramatic results, including stable expression of functional beta-globin resulting in transfusion independence which now extends for greater than four years following a single treatment.
This CIRM award is among the first awards under the agency’s Strategic Partnership Awards initiative, which is designed to engage more effectively with industry and to increase outside investment in CIRM-funded stem cell research. The funding awards were made at the October 25, 2012 meeting of the stem cell agency’s governing board, the Independent Citizens Oversight Committee (ICOC).
CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was overwhelmingly approved by voters, and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. A list of grants and loans awarded to date may be seen here: http://www.cirm.ca.gov/for-researchers/researchfunding.
About bluebird bio
bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bio’s product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The company’s novel approach uses stem cells harvested from the patient’s own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bio’s approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and potentially presenting a one-time transformative therapy using a patient’s own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease. Led by a world-class team, its operations are located in Cambridge, Mass., San Francisco and Paris, France. For more information, please visit www.bluebirdbio.com.