- Sangamo Therapeutics and Novartis have agreed to work together to develop treatments that aim to alter the expression of key genes implicated in neurodevelopmental disorders like autism.
- Novartis will pay Sangamo $75 million within 30 days and could add as much as $720 million more if the target drugs hit future development and commercial milestones. In return, the Swiss drugmaker will get exclusive rights to gene editing treatments aimed at three undisclosed genes.
- The agreement is the latest in a string of partnerships for Sangamo, which is starting to capitalize on growing interest by larger firms in using genetic medicine to go after tough-to-treat neurological diseases. Sangamo struck a wide-ranging deal with Biogen earlier this year.
The deal suggests the appeal of Sangamo's technology, which use zinc finger proteins to turn genes on or off.
The agreement with Novartis marks the sixth collaboration Sangamo has signed with a major pharmaceutical partner, and the second of late to be focused exclusively on brain diseases. Earlier this year, Biogen agreed to pay the California biotech $350 million upfront and as much as $2.4 billion in conditional payments as part of a deal focused on neurological conditions like Alzheimer's disease.
By partnering, Novartis and Sangamo hope to marry two different approaches to genetic medicine in an effort to go after diseases that traditional drugs haven't been able to address.
Novartis, through its buyout of AveXis, has extensive experience with gene therapy. AveXis develops treatments that use adeno-associated viruses to transport genes to specific targets in the body. One of them, Zolgensma, is approved in the U.S., Europe and Japan to treat spinal muscular atrophy.
But the AAV technology Novartis uses can't easily shuttle large genes into the body. That's one of the reasons, for instance, several AAV-based gene therapies in development for Duchenne muscular dystrophy use a shortened version of the gene that's defective in the disease.
Novartis believes Sangamo's technology can solve that type of problem, and aims to go after larger genes implicated in neurodevelopment diseases. Sangamo's zinc finger proteins are small enough to fit into an AAV.
"This dramatically expands the range of diseases that we can potentially target with gene therapy because many diseases are caused by the loss of a single copy of a gene," said Ricardo Dolmetsch, head of neuroscience at the Novartis Institutes for BioMedical Research, in a blog post on the partnership.
In targeting autism spectrum disorder, the companies are trying to tackle a condition that may affect as many as one in every 59 children, impairing their social interaction, communication and daily activity. It's most often treated with behavioral and educational therapies.
Novartis didn't specify the other conditions that will be part of the collaboration, only saying the team will work to identify treatments for several diseases. Under the deal, the company also has the right to license proprietary AAVs that Sangamo has been developing.