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Genentech-Sangamo deal to advance neurodegenerative treatments

Overall, Sangamo Therapeutics will be eligible up to $1.9 billion from Genentech, which includes various milestone and royalty payments.

Genentech Neurodegenerative Diseases

Credit: Michael Vi / Shutterstock.com

Genentech, a member of the Roche Group, and Sangamo Therapeutics have agreed to work together to develop intravenous genomic medicines for neurodegenerative diseases.

Genentech will gain rights to Sangamo’s proprietary zinc finger repressors, which are “directed to the tau gene” and play a vital role in Alzheimer’s disease and other tauopathies”

Sangamo is eligible for a total of up to $1.9 billion under the agreement. This is subject to specific conditions including achievement of development and commercial milestones across numerous potential products. The deal also involves Genentech agreeing to pay Sangamo $50 million, which includes milestone payments.

Genentech will gain rights to Sangamo’s proprietary zinc finger repressors, which are “directed to the tau gene” and play a vital role in Alzheimer’s and other tauopathies, Sangamo stated.

Further details of the license agreement

Terms of the deal also gives Genentech an exclusive license to Sangamo’s proprietary, neurotropic adeno-associated virus (AAV) capsid, STAC-BBB. Pre-clinical trials in nonhuman primates have shown its ability to penetrate the blood-brain barrier and achieve brain transduction, according to Sangamo.

The company’s “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,” Sandy Macrae, the firm’s Chief Executive Officer commented.

Furthermore, as part of the transaction, Genentech is responsible for “clinical development, regulatory interactions, manufacturing and global commercialisation”. Sangamo shared that it will oversee technology transfer and certain preclinical activities.

Advancing other treatments for neurodegenerative diseases

In June, Genentech shared promising five-year data from the company’s extension study of the pivotal Phase II FIREFISH clinical trial evaluating Evrysdi® (risdiplam) in children with Type 1 spinal muscular atrophy (SMA).