Novartis Enters $75 Million Collaboration with Sangamo for its Zinc Finger Gene Editing Technology

by Ruchi Jhonsa
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By Ruchi Jhonsa, Ph.D.

Just months after Sangamo Therapeutics, a genomic medicine company struck a neurological disease deal with Biogen, it made a second deal today with pharma giant Novartis for three neurodevelopmental disorders, including intellectual disability and autism spectrum disorder (ASD). This deal combines Novartis’ experience in neuroscience drug development with Sangamo’s proprietary zinc finger gene-editing technology to tackle neurodevelopmental disorders.

Under the agreement, Novartis will hand over $75 million upfront and an additional $720 million in milestone payments over three years to Sangamo. Additionally, Sangamo is eligible to receive single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration. For Novartis, the deal fits squarely with its neuroscience focus and expands upon its existing gene therapy investments. The gene therapy Zolgensma, developed by the company, got the USFDA approval last year and has received conditional approval in Europe for the treatment of spinal muscular atrophy.

Sangamo is a pioneer in the development of zinc finger nucleases (ZFNs), which, like CRISPR, can cut precise sequences of DNA for gene editing. In 2018, the company attempted gene editing directly in the human body, making it the first company ever to do so in humans. The company’s deal with Novartis, however, highlights a different use of zinc fingers. It combines Zinc fingers that can recognize but not cut DNA with transcription factors that turn a gene on or off. The combination is a zinc finger protein transcription factor (ZFP-TFs) that will be used by Novartis to shut off proteins implicated in neurological diseases.

The genes encoding ZFP-TFs are delivered into the brain or spinal cord by AAV viral vectors. The company is currently developing new variants of the AAV delivery vehicle that can reach different parts of the brain affected by various neurological disorders.

“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 of bringing 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 Sandy Macrae, CEO of Sangamo.

“Partnering Sangamo’s proprietary technology with Novartis’ deep experience in neuroscience drug development is a powerful combination which expands Sangamo’s pipeline and allows us to tackle challenging neurodevelopmental conditions. Our goal in this collaboration is to create genomic medicines for patients with neurodevelopmental disorders, such as autism, that can potentially alter the natural history of these complex lifelong disorders,” he added.

Over a three-year collaboration period, the duo will work on the development of ZFP-TFs targeted to three undisclosed genes associated with neurodevelopmental disorders, including ASD and intellectual disability, to which Novartis will have exclusive rights. While Sangamo will put efforts into certain research and related manufacturing activities, Novartis will work on additional research activities, investigational new drug-enabling studies, clinical development, regulatory interactions, manufacturing, and global commercialization and will fund all the research and manufacturing activities conducted by Sangamo related to the three targets. The deal also grants Novartis the option to license Sangamo’s proprietary AAVs.

“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.”

Related Article: Merck and Yumanity Sign $500 Million Deal to Develop ALS and FTLD Treatments



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