Biogen Idec and Sangamo BioSciences have entered an exclusive worldwide collaboration and license agreement to develop therapeutics for hemoglobinopathies, inherited conditions resulting abnormal structure or underproduction of hemoglobin. Biogen will employ its expertise in non-malignant hematology by leveraging Sangamo's zinc finger nuclease (ZFN) genome-editing technology platform to develop treatments targeting sickle cell disease (SCD) and beta-thalassemia. The ZFN technology can be used to precisely target and knock out key regulators of gene expression, or insert a corrective gene to replace the defective copy.
Sangamo is responsible for all R&D activities through clinical proof of concept in beta-thalassemia, and both companies will contribute to the submission of an IND application for SCD. Biogen Idec will be responsible for worldwide clinical development and commercialization of any resulting products. Sangamo retains an option to co-promote any licensed product in the U.S.
Sangamo will receive an upfront payment of $20 million and will be reimbursed for R&D-related costs. Sangamo is also eligible to receive approximately $300 million based on certain development, regulatory, commercialization, and sales milestones, as well as royalties.
"Our collaboration with Sangamo is expected to help us expand our capabilities to develop treatments for people with serious, inherited hematologic conditions," said Douglas E. Williams, Ph.D., Biogen Idec's executive vice president of R&D. "Building upon emerging science related to fetal hemoglobin regulation, we intend to develop Sangamo's novel gene-editing technology to create a single approach that has the potential to functionally cure both sickle cell disease and beta-thalassemia."