Novartis and Sangamo Therapeutics, a Brisbane, California-based genomic medicine company, have formed a global licensing collaboration agreement, worth up to $795 million, to develop and commercialize gene-regulation therapies to address three neurodevelopmental targets, including those for autism spectrum disorder (ASD) and other neurodevelopmental disorders.

The collaboration will use Sangamo’s genome-regulation technology, zinc finger protein transcription factors (ZFP-TFs), which are delivered with adeno-associated viruses (AAVs), to upregulate, or activate, the expression of genes that are inadequately expressed in individuals with certain types of neurodevelopmental disorders.

Under the agreement, Novartis will pay Sangamo a $75-million upfront license-fee payment within 30 days. In addition, 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.

In addition, over a three-year collaboration period, 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.

Source: Sangamo Therapeutics