Sangamo and Novartis partner on gene therapies for autism

By Ben Hargreaves

- Last updated on GMT

© Tetiana Lazunova / Getty Images
© Tetiana Lazunova / Getty Images

Related tags Sangamo Therapeutics Novartis Gene therapy autism

The two companies will develop three neurodevelopmental targets, using the former’s zinc-finger technology.

The targets will be three undisclosed genes that are associated with certain neurodevelopmental disorders, such as autism spectrum disorder (ASD) and intellectual disability.

Sangamo Therapeutics will bring its genome regulation technology to upregulate the expression of key genes involved in neurodevelopmental disorders.

To gain access to this technology, Novartis will pay $75m (€63m) upfront to Sangamo. Beyond this, Novartis could end up paying an additional $720m in developmental milestones.

According to Sangamo, the hope is that its zing-finger technology can active the expression of genes that are inadequately expressed in individuals with certain types of neurodevelopmental disorders.

Sangamo’s potentially therapy is currently delivered through adeno-associated viruses (AAVs) to the DNA level.

The terms of the agreement see the collaboration playing out over a three-year period, with Novartis holding the exclusive rights to zinc finger protein transcription factors (ZFP-TFs) targeting the three undisclosed genes at the center of the deal.

In addition, Novartis will have the option to license Sangamo proprietary AAVs delivery technology.

In terms of development, Sangamo will remain in the lead of research and associated manufacturing activities, though with funding from Novartis, and the latter company will perform supplementary research activities, before stepping in to take the lead in regulatory and commercial activities later in development.

Jay Bradner, president of the Novartis Institutes for BioMedical Research, said, “The goal [of the partnership] 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.”

Gene therapy has become a focus area for Novartis over the last few years, as the company invests into its cell and gene manufacturing capabilities​ and opens new treatment avenues​ for patients with genetic conditions.

Alongside these new developments, Novartis has also called for ‘rethink’ of how healthcare is costed and what the value such treatments provide to society are, a debate that has come to the fore as the issue of the pricing of such therapies has arisen​.

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