Genentech Enters an Approximately $2 Billion Deal to License Sangamo Therapeutics Technologies for Developing Neurodegenerative Disease Therapeutics

Editor's note: this story was originally published on BioPharmInternationalcom.

Genentech, a member of the Roche Group, has entered into a license agreement worth nearly up to $2 billion potentially, with Sangamo Therapeutics, a genomic medicine company. Through the deal, Genentech aims to develop genomic medicines for treating certain neurodegenerative diseases via intravenous administration. Sangamo is giving Genentech an exclusive license to its proprietary zinc finger repressors directed to the tau gene and its neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, for tau and a second neurology target.

Under the agreement, Sangamo will be responsible for completing a technology transfer to Genentech and will be responsible for certain preclinical activities. For its part, Genentech will be responsible for all clinical development, regulatory interactions, manufacturing, and global commercialization. Genentech expects to pay Sangamo $50 million in near-term upfront license fees and milestone payments. In addition, Sangamo is eligible to receive up to $1.9 billion in development and commercial milestones. These milestone payments would be spread across multiple potential products developed under the agreement. Sangamo is also eligible to receive tiered royalties on net sales of any products resulting from the collaboration, subject to certain specified reductions.

“Sangamo has been pioneering the field of genomic medicine for years to address devastating neurodegenerative diseases with limited current treatment options,” said Sandy Macrae, CEO, Sangamo, in the press release. “We strongly believe in the power of our zinc finger technology to regulate the expression of key genes involved in disease. The recent discovery of our industry-leading intravenously delivered AAV capsid, STAC-BBB, has the potential to address longstanding challenges in delivering therapeutics to the central nervous system. We are excited to share this powerful combination with Genentech to advance potential treatment options for devastating neurodegenerative disorders, and we are hopeful this could be the first of multiple capsid collaborations to come with other partners.”

The tau gene is critically involved in Alzheimer’s disease (AD) and other tauopathies (1). Genentech’s access to Sangamo’s zinc finger repressors will allow Genentech to develop genomic therapeutics against AD as well as an undisclosed second neurology target. Sangamo’s STAC-BBB has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates, the company said in the release.

“We are uniquely positioned with our collective experience, expertise, and resources in neurological research to explore transformative approaches, including gene therapy, that treat neurodegenerative diseases,” said Boris L. Zaïtra, head of Roche Corporate Business Development, in the release. “Through groundbreaking research and partnerships with companies such as Sangamo, we are committed to pursuing important breakthroughs in both early diagnosis and treatment. Our relentless pursuit of scientific innovation is taking us into areas of enormous unmet medical need and progress in treating diseases of the brain and nervous system.”

Reference

1. Medeiros, R.; Baglietto-Vargas, D.; LaFerla, F. M. The Role of Tau in Alzheimer's Disease and Related Disorders. CNS Neurosci. Ther. 2011, 17 (5), 514–524. DOI: 10.1111/j.1755-5949.2010.00177.x

Source: Sangamo Therapeutics