Sangamo Therapeutics, Inc., a genomic medicine company, announced it has entered into a license agreement with Genentech, a member of the Roche Group, to develop intravenously administered genomic medicines to treat certain neurodegenerative diseases. Sangamo has granted Genentech an exclusive license to Sangamo’s proprietary zinc finger repressors that are directed to the tau gene, which is critically involved in Alzheimer’s disease and other tauopathies, as well as an undisclosed second neurology target. Sangamo has also agreed to exclusively license to Genentech, for tau and the second neurology target, Sangamo’s proprietary, neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates.
“Sangamo has been pioneering the field of genomic medicine for years to address devastating neurodegenerative diseases with limited current treatment options,” said Sandy Macrae, Chief Executive Officer of Sangamo. “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.”
“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. “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.”
Under the terms of the agreement, Sangamo is responsible for completing a technology transfer and certain preclinical activities, and Genentech is responsible for all clinical development, regulatory interactions, manufacturing and global commercialization. Genentech is expected to pay Sangamo $50 million in near-term upfront license fees and milestone payments. Sangamo is eligible to earn up to $1.9 billion in development and commercial milestones spread across multiple potential products under the agreement and tiered royalties on net sales of such products, subject to certain specified reductions.
Sangamo continues to engage in business development discussions with additional potential collaboration partners about the Sangamo STAC-BBB capsid delivery platform, its epigenetic regulation capabilities, and other assets, including Fabry disease.