Despite decades of research and many millions of dollars invested, Roche still has yet to develop a drug that works against Alzheimer’s disease. The Swiss pharmaceutical giant hasn’t given up, though, and on Tuesday announced a new deal that offers another shot at its enduring goal.
Through its Genentech research arm, Roche has licensed technology from Sangamo Therapeutics and plans to use it to create genomic medicines for select neurodegenerative diseases. Specifically, the agreement grants exclusive rights to Sangamo’s “zinc finger” molecules designed to repress the gene that makes “tau,” a protein many scientists view as a main driver of Alzheimer’s.
Roche now also has a license to molecules aimed at a second, undisclosed neurology target, as well as a microscopic shuttle that in primate studies was able to cross the blood-brain barrier — a major obstacle in brain drug development. Sangamo recently discovered this shuttle, an adeno-associated virus capsid, and believes it “has the potential to address longstanding challenges in delivering therapeutics to the central nervous system,” according to Sandy Macrae, the company’s CEO.
Sangamo is “hopeful this could be the first of multiple capsid collaborations to come with other partners,” Macrae said in a statement.
Genentech heavily backloaded the deal, which has a total value of nearly $2 billion. Sangamo will receive $50 million in upfront licensing fees and near-term milestone payments. But it could take home up to $1.9 billion more if its medicines hit certain development and commercial goals. The California-based biotechnology company would also receive tiered royalties on net sales of any products stemming from the collaboration.
Per deal terms, Sangamo is responsible for some preclinical activities while Genentech will take the lead on all clinical development, regulatory interactions, manufacturing and global commercialization.
For Sangamo, the new partnership provides a welcome source of cash. The company last year culled its research pipeline and twice turned to layoffs as a way to lower expenses. The second of those layoff rounds affected 40% of Sangmo’s workforce.
As of March 31, the biotech had around $54 million in cash and cash equivalents.
In the Tuesday statement, Sangamo said it “continues to engage in business development discussions” with potential partners for its various technologies and assets, including an experimental gene therapy for a rare neurological disorder called Fabry disease.