Sangamo Therapeutics has signed a global licence agreement with Roche Group member Genentech for the development of genomic medicines to treat neurodegenerative diseases.
The collaboration focuses on intravenously administered treatments targeting the tau gene, associated with Alzheimer's disease and other tauopathies, and a second undisclosed neurology target.
Sangamo has granted Genentech an exclusive licence for its zinc finger repressors for the tau gene and the neurology target.
Sangamo's neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, will be exclusively licensed to Genentech for these targets.
STAC-BBB has shown significant blood-brain barrier penetration and brain transduction in nonhuman primates.
Sangamo is tasked with concluding a technology transfer and certain preclinical works, while Genentech will handle the complete clinical development, regulatory works, manufacture and commercialisation of the assets globally.
Sangamo is entitled to receive $50m from Genentech in near-term upfront license fees and milestone payments, and could receive $1.9bn in development and commercial milestones across multiple potential products, as well as tiered royalties on net product sales.
The company is also in discussions with other collaboration partners regarding its STAC-BBB capsid delivery platform, epigenetic regulation capabilities and other assets, including treatments for Fabry disease.
Sangamo Therapeutics CEO Sandy Macrae stated: “Sangamo has been pioneering the field of genomic medicine for years to address devastating neurodegenerative diseases with limited current treatment options.
“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.”
In January 2024, Genentech partnered with GenEdit to use the latter company’s non-viral delivery platform for gene editing therapies.
