Sangamo Announces Global Collaboration With Novartis to Develop Genomic Medicines for Autism and Other Neurodevelopmental Disorders
- Collaboration for gene regulation therapies for three neurodevelopmental targets, including for genes linked to autism spectrum disorder and intellectual disability
- Sangamo will receive a
“At Sangamo, we believe that we can engineer zinc finger proteins to address virtually any genomic target, and we are building a broad pipeline of wholly owned and partnered programs with the goal to bring our genomic medicines to patients. In the case of the central nervous system, there are potentially hundreds of neurological disease gene targets that may be addressable by our zinc finger platform,” said
“This collaboration with Sangamo is part of our commitment to pioneering the next generation of neurodevelopmental treatments,” said
Sangamo’s ZFP-TF genome regulation technology, which is currently delivered with adeno-associated viruses (AAVs), functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. The collaboration will leverage ZFP-TFs engineered by Sangamo scientists in an effort to upregulate, or activate, the expression of genes that are inadequately expressed in individuals with certain types of neurodevelopmental disorders.
Under the terms of the agreement, over a three-year collaboration period, Novartis has exclusive rights to ZFP-TFs targeted to three undisclosed genes which are associated with neurodevelopmental disorders, including ASD and intellectual disability. Novartis also has the option to license Sangamo’s proprietary AAVs. Sangamo is responsible for certain research and associated manufacturing activities, all of which will be funded by Novartis, and Novartis assumes responsibility for additional research activities, investigational new drug-enabling studies, clinical development, related regulatory interactions, manufacturing and global commercialization.
Under the collaboration agreement, Novartis will pay Sangamo a
Forward Looking Statements
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize safe and effective therapies to treat neurodevelopmental disorders and the timing and availability of such therapies, the potential for Sangamo to receive upfront licensing fees and earn milestone payments and royalties under the Novartis collaboration and the timing of such fees, payments and royalties, the potential to use ZFP-TF technology to upregulate specific genes involved with neurodevelopmental disorders, the therapeutic potential of Sangamo’s zinc finger platform and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the evolving COVID-19 pandemic and its impact on the global business environment, healthcare systems and the business and operations of Sangamo and Novartis; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products and product candidates; the commercialization of approved products; the potential for technological developments that obviate technologies used by Sangamo and Novartis; the potential for Novartis to breach or terminate the collaboration agreement; and the potential for Sangamo to fail to realize its expected benefits of the Novartis collaboration. There can be no assurance that Sangamo will earn any milestone or royalty payments under the Novartis collaboration or obtain regulatory approvals for product candidates arising from these collaborations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's and Novartis’ operations and business environments. These risks and uncertainties are described more fully in Sangamo's filings with the
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