Roche's Genentech has signed a license agreement with Sangamo Therapeutics to develop intravenous genomic medicines to treat certain neurodegenerative conditions, including Alzheimer’s disease.
The California-based genomic medicines company has granted Genentech an exclusive license to its epigenetic regulation capabilities — 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. According to Sangamo, zinc finger proteins are naturally occurring proteins in humans which can recognize and bind to specific DNA sequences. Their natural function is to activate or repress the expression of human genes by turning them on or off.
Sangamo has also agreed to exclusively license its neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, to Genentech for tau and the second neurology target. STAC-BBB has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates.
Per the deal, Sangamo is responsible for completing a tech transfer and certain preclinical activities, and Genentech is responsible for all clinical development, regulatory interactions, manufacturing and global commercialization. Genentech will 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.
The deal is good news for Sangamo, after Novartis and Biogen both walked away from separate neurodegenerative deals last year, leaving Sangamo scrambling to assess its options. The Novartis deal, which involved a $75 million upfront license fee and up to $720 million in milestone payments, had focused on leveraging Sangamo’s zinc finger protein transcription factors against three neurodevelopmental targets, including autism spectrum disorder. The Biogen deal, which involved an upfront payment of $350 million with Sangamo eligible to receive up to $2.37 billion in milestones, focused on the development of gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases.
In April 2023, a month after Novartis and Biogen bailed on the deals, Sangamo announced a strategic pipeline prioritization and corporate restructuring, including a workforce reduction of approximately 27%. The company regrouped with a plan to focus on three key areas: Nav 1.7 and Prion as cornerstones to the neurology epigenetic regulation portfolio; Fabry phase 3 readiness; and its TX200 CAR-Treg clinical study.