Sangamo Enters Genomic Medicines Collaboration with Novartis for Autism and Other Neurodevelopmental Disorders

Rare Daily Staff

Sangamo Therapeutics said it has executed a global licensing collaboration agreement with Novartis to develop and commercialize gene regulation therapies to address three neurodevelopmental targets, including autism spectrum disorder and other neurodevelopmental disorders for $75 million upfront.

Under the collaboration agreement, Sangamo is eligible to earn up to $720 million in other development and commercial milestone payments, including up to $420 million in development milestones and up to $300 million in commercial milestones. Sangamo is also eligible to receive from Novartis tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration.

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.

“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,” said Sandy Macrae, CEO of Sangamo. “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.”

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.

Under the terms of the three-year agreement, Novartis has exclusive rights to ZFP-TFs targeted to three undisclosed genes that 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.

“This collaboration with Sangamo is part of our commitment to pioneering the next generation of neurodevelopmental treatments,” said Jay Bradner, president of the Novartis Institutes for BioMedical Research. “The goal is to create new gene regulation therapies that act at the genomic level, moving us beyond the symptom focused treatments of today and toward therapies that can address some of the most challenging neurodevelopmental disorders.”

Photo: Sandy Macrae, CEO of Sangamo