Biogen Idec and Sangamo BioSciences said today they launched a collaboration that will apply the latter’s genome-editing technology platform to develop treatments targeting sickle cell disease (SCD) and beta thalassemia, in a deal that could net Sangamo up to about $320 million and more.
Under their exclusive worldwide collaboration and license agreement, Biogen Idec will pay Sangamo $20 million up front; up to $300 million in payments tied to development, regulatory, commercialization, and sales milestones; as well as double-digit royalties on product sales.
In return, Sangamo will oversee all research and development activities through the first clinical proof of concept trial in beta thalassemia, while both companies will perform activities designed to enable submission of an IND application for SCD. Biogen Idec will be responsible for subsequent worldwide clinical development and commercialization of products arising from the alliance, though Sangamo retains an option to co-promote any licensed product to treat SCD and beta thalassemia in the United States.
“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 evp of research and development. “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 SCD and beta thalassemia.”
The companies said the deal has been approved by their boards of directors, and is subject to customary closing conditions including expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the United States.
The collaboration will use Sangamo’s zinc finger nuclease (ZFN) genome-editing technology, designed to enable multiple approaches to treating SCD and beta thalassemia. Both diseases manifest after birth, when patients switch from producing functional fetal gamma-globin to a mutant form of adult beta-globin. ZFN can precisely target and disrupt key transcriptional regulators to reverse the switch from expression of the mutant adult beta-globin back to the production of functional fetal gamma-globin. Or the technology can be used to precisely insert a new corrected beta-globin gene to replace the defective copy.
By engineering a class of DNA-binding proteins called zinc finger DNA-binding proteins (ZFPs) that can recognize a specific DNA sequence, Sangamo has created sequence-specific ZFNs for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function.
Sangamo has ongoing Phase II and Phase I/II clinical trials to assess the safety and efficacy of a new ZFP Therapeutic® for HIV/AIDS, as well as a fully enrolled and funded Phase II trial to evaluate NGF-AAV (CERE-110) in Alzheimer’s disease, a program the company acquired as part of its purchase of Ceregene.
The Biogen Idec alliance “is further validation of our ZFP platform as a transformative technology and accelerates our goal of developing a novel class of therapeutics which has the potential to revolutionize the treatment of genetic diseases,” Edward Lanphier, Sangamo’s president and CEO, said in a statement.
Sangamo is also applying its technologies in a separate collaboration with Shire to develop new drugs for hemophilia, Huntington’s disease, and other monogenic diseases, as well as through partnerships with companies in nontherapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich.