Genentech, part of Roche, has signed an exclusive license with Sangamo Therapeutics to develop genomic treatments for two neurodegenerative diseases. Sangamo will receive $50 million up front and could earn up to $1.9 billion in development and commercial milestone payments plus tiered royalties for its proprietary zinc finger repressors directed to the gene encoding the tau protein, which is involved in Alzheimer’s disease. The deal also includes Sangamo’s neurotropic adeno-associated virus (AAV) capsid delivery system, which has demonstrated potent blood–brain barrier penetration and brain transduction in nonhuman primates. Genentech will use it to target tau and another undisclosed neurology target.
“We strongly believe in the power of our zinc finger technology to regulate the expression of key genes involved in disease,” said Sangamo CEO Sandy Macrae. “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.”
