Genentech and Sangamo Forge $1.95B Collaboration to Develop Intravenous Gene Therapies for Neurodegenerative Diseases

Aug 06, 2024 21:55 CST Updated 21:55

Genentech

Pharmaceutical R&D Manufacturer

On August 6, Richmond, California, genomic medicine company Sangamo Therapeutics announced that it has reached an agreement with Roche's subsidiary,Genentech (Genentech) to sign a licensing agreement to develop intravenous genomic drugs for the treatment of certain neurodegenerative diseases. Sangamo has granted Genentech access to Sangamo's proprietary zinc finger inhibitors (zinc finger repressors) for exclusive licensing, these inhibitors target the tau gene, which is closely associated with Alzheimer's disease and other tauopathies, as well as an undisclosed second neurological target. Sangamo also agreed toGenentechExclusive license for tau and a second neurological target, Sangamo’s proprietary neurotropic adeno-associated virus (AAV) capsid STAC-BBB, which has demonstrated robust blood-brain barrier penetration and brain transduction in non-human primates.

Sangamo CEO Sandy Macrae stated:"For years, Sangamo has been at the forefront of genomic medicine to address devastating neurodegenerative diseases with currently limited treatment options. We firmly believe in the power of our zinc finger technology to modulate the expression of key disease-related genes. We have recently discovered STAC-BBB, an industry-leading intravenously injectable AAV capsid, which has the potential to overcome long-standing challenges in central nervous system therapeutics. We are excited to share this powerful combination with Genentech to advance potential treatment options for devastating neurodegenerative diseases, and we hope this may be the first of multiple capsid collaborations with other partners."

Boris L. Zaïtra, Head of Roche Business Development, said:"We have a unique collective experience, expertise, and resources in neuroscience research to explore transformative approaches to treating neurodegenerative diseases, including gene therapy. Through groundbreaking research and collaborations with companies like Sangamo, we are committed to making significant breakthroughs in early diagnosis and treatment. Our relentless pursuit of scientific innovation has led us into areas of great unmet medical need, making progress in the treatment of brain and nervous system disorders."

Under the terms of the agreement, Sangamo is responsible for completing the technology transfer and certain preclinical activities, while Genentech will handle all clinical development, regulatory interactions, manufacturing, and global commercialization. Genentech is expected to pay Sangamo $50 million in short-term upfront licensing fees and milestone payments. According to the agreement, Sangamo is eligible to receive up to $1.9 billion in development and commercial milestones for multiple potential products, along with tiered royalties on net sales of these products, subject to certain specific reductions.

Sangamo continues business development discussions with other potential partners regarding the STAC-BBB capsid delivery platform, its epigenetic regulation capabilities, and other assets, including Fabry disease.

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