Genentech, a subsidiary of the Roche Group, has entered into a significant licensing agreement with Sangamo Therapeutics, potentially valued at nearly $2 billion. This deal is set to advance the development of genomic medicines aimed at treating certain neurodegenerative diseases through intravenous administration. Under the terms of the agreement, Genentech will gain exclusive rights to Sangamo’s proprietary technologies, including zinc finger repressors targeting the tau gene and the novel neurotropic adeno-associated virus (AAV) capsid known as STAC-BBB.
Sangamo Therapeutics will handle the technology transfer to Genentech and perform specific preclinical activities. Conversely, Genentech will oversee all aspects of clinical development, regulatory interactions, manufacturing, and global commercialization of the resulting therapies. The initial phase of the agreement includes a $50 million upfront payment from Genentech, with the potential for Sangamo to earn up to $1.9 billion in development and commercial milestones. Additionally, Sangamo stands to receive tiered royalties on net sales from any products developed through this collaboration, though these royalties are subject to certain reductions.
Sangamo’s CEO, Sandy Macrae, emphasized the groundbreaking nature of their genomic medicine technologies. He noted that the company’s zinc finger technology has the potential to regulate the expression of crucial genes involved in neurodegenerative diseases. Particularly, the STAC-BBB AAV capsid is designed to overcome challenges associated with delivering therapeutics across the blood-brain barrier, a key hurdle in treating central nervous system disorders. Macrae expressed enthusiasm about the partnership with Genentech, anticipating that it could pave the way for further collaborations involving their advanced capsid technology.
The tau gene, which is central to Alzheimer’s disease and other tauopathies, will be a primary target in this collaboration. Genentech’s access to Sangamo’s technology will enable the development of genomic therapeutics aimed at Alzheimer’s disease and potentially another undisclosed neurological target. The STAC-BBB capsid has demonstrated significant efficacy in penetrating the blood-brain barrier and achieving brain transduction in preclinical studies with nonhuman primates, indicating promising prospects for therapeutic delivery.
Boris L. Zaïtra, head of Roche Corporate Business Development, highlighted the strategic importance of this partnership in Roche’s broader commitment to exploring transformative treatments for neurodegenerative diseases. Zaïtra underscored the collective expertise and resources that both companies bring to the table, emphasizing their shared goal of pursuing innovative breakthroughs in the diagnosis and treatment of brain and nervous system diseases.
Reference:
- Medeiros, R.; Baglietto-Vargas, D.; LaFerla, F. M. The Role of Tau in Alzheimer’s Disease and Related Disorders. CNS Neurosci. Ther. 2011, 17 (5), 514–524. DOI: 10.1111/j.1755-5949.2010.00177.x
