Genomic medicine company Sangamo Therapeutics announced today that it has entered a global licensing collaboration agreement with Novartis to develop and commercialize gene regulation therapies for three neurodevelopmental targets, including autism spectrum disorder.
Genomic medicine company Sangamo Therapeutics announced today that it has entered a global licensing collaboration agreement with Novartis to develop and commercialize gene regulation therapies for three neurodevelopmental targets, including autism spectrum disorder.
Sangamo’s proprietary genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), will be leveraged under the agreement to upregulate the expression of key genes in neurodevelopmental 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 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. 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.”
Sangamo’s ZFP-TF genome regulation technology is currently delivered with adeno-associated viruses (AAVs). It functions at the DNA level to selectively repress or activate the expression of specific genes for a therapeutic effect.
“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.”
According to the terms of the collaboration agreement, Novartis will pay Sangamo a $75 million upfront license fee payment within 30 days. Sangamo is also eligible to earn up to $720 million in other development and commercial milestone payments.
This is not the only agreement that Sangamo has entered as of late. In April, the company announced that it had entered a collaboration and exclusive license agreement with Mogrify Ltd. Under the terms, Sangamo is working to develop allogenic cell therapies from Mogrify’s proprietary induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs), and Sangamo’s zinc finger protein gene-engineered chimeric antigen receptor regulatory T cell (CAR-Treg) technology.
Mogrify is responsible for the discovery and optimization of the cell conversion technology from iPSCs or ESCs to regulatory T cells. Sangamo has been granted exclusive rights to use Mogrify’s technology to create Tregs from iPSCs or ESCs. Afterward, the company may then use its ZFP gene-engineering technology and therapeutic development capabilities to transform the Tregs into novel allogenic CAR-Treg cell therapy candidates. Ultimately, the goal is to take these candidates through clinical development for the treatment of inflammatory and autoimmune diseases.
Sangamo will pay Mogrify an upfront payment, and Mogrify is also eligible to receive potential additional payments related to development and regulatory milestones.
“This license agreement provides Sangamo with access to Mogrify’s cell conversion technology, which will diversify our options as we develop off-the-shelf allogeneic CAR-Treg cell therapies,” said Jason Fontenot, SVP, Head of Cell Therapy at Sangamo. “We expect this collaboration to accelerate our development of scalable and accessible CAR-Treg cell therapies, so that we can potentially deliver treatments to patients with inflammatory and autoimmune diseases more rapidly.”
