Sangamo Therapeutics has announced a licensing agreement worth up to $1.95bn with Genentech, a member of the Roche Group, to develop intravenous genomic therapies for specific neurodegenerative diseases.
Under the agreement, Sangamo Therapeutics is granting Genentech an exclusive license to its proprietary zinc finger repressors targeting the tau gene and a second, undisclosed neurology target.
Additionally, the genomic medicine firm will provide Genentech with an exclusive license for its proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB.
The AAV capsid has shown its ability to penetrate the blood-brain barrier and transduce brain tissue in non-human primates.
Sangamo Therapeutics CEO Sandy Macrae said: “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.”
As per the terms of the agreement, the genomic medicine company will handle the technology transfer and complete certain preclinical activities, while Genentech will take on responsibilities for clinical development, regulatory affairs, manufacturing, and global commercialisation.
In return, Genentech will pay $50m in upfront license fees and milestone payments to Sangamo Therapeutics.
Additionally, Sangamo Therapeutics could earn up to $1.9bn in development and commercial milestones across various potential products, along with tiered royalties on net sales of these products, subject to certain reductions.
Roche corporate business development head Boris Zaïtra said: “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.”
Furthermore, Sangamo Therapeutics is actively pursuing business development opportunities with other potential partners to explore collaborations related to its STAC-BBB capsid delivery platform, epigenetic regulation capabilities, and other assets.
The latest deal comes after Pfizer announced positive topline results from Phase 3 AFFINE study of investigational haemophilia A gene therapy candidate, giroctocogene fitelparvovec.
Sangamo Therapeutics is co-developing this potential gene therapy with Pfizer and licensing it to the latter as per a deal signed in late 2019.
