genOway acquired an exclusive worldwide license, and sublicensing rights, to bluebird bio’s RMCE technology for generating genetically modified rodents. The recombinase-based technology, developed at the Massachusetts Institute of Technology, has been designed to enable the insertion of gene sequences at specified loci.
genOway is dedicated to the design, development, and marketing of genetically modified biological models and associated technologies. The firm says it has already received considerable interest in the RMCE platform from the pharma industry, “in particular since we will guarantee our licensees with a total freedom to operate for their R&D activities,” comments CEO Alexandre Fraichard. “This technology represents a clear competitive advantage by strengthening our proprietary technology portfolio and increasing the uniqueness of our commercial offering.”
Bluebird is focused on developing autologous bone marrow stem cell-based gene therapies for severe genetic disorders. The firm has two clinical-stage products: a gene therapy for childhood adrenoleukodystrophy (CCALD), and its LentiGlobin® program against thalassemia and sickle cell anemia. Adrenoleukodystrophy is a rare, inherited neurological disorder that in its most severe form causes damage to the myelin sheath and progressive dysfunction of the adrenal glands.
Data from a pilot study with bluebird’s CCALD gene therapy, reported in 2010, demonstrated continued stable expression of the CCALD protein and disease stabilization at three years in two CCALD patients. The firm says it plans to start larger clinical studies in the U.S. and Europe during 2011.
The LentiGlobin program, currently undergoing Phase I/II evaluation, is designed to deliver a fully functional human beta-globin gene, under the control of the beta-globin promoter and locus control regions, to the patient’s own hematopoietic stem cells.