Novartis has commercialized a gene therapy that targets the central nervous system, but the pharma giant has more CNS gene therapies in its pipeline, and it’s looking to improve how those therapies reach their goals. Voyager Therapeutics has the technology to find new ways to efficiently deliver gene therapy into the brain.Novartis sees promise in Voyager platform and has agreed to fund more than $54 million to start a project Multi-Therapeutic Alliance.
Most gene therapy is delivered by adeno-associated virus. Genetic cargo is carried inside a protein shell called the capsid. Novartis’ first gene therapy, spinal muscular atrophy treatment Zolgensma, uses an AAV vector to cross the blood-brain barrier and enter motor neuron cells, where the capsid releases its genetic payload.
Voyager’s proprietary technology, TRACER, has discovered novel AAV capsids capable of delivering gene therapy to specific types of tissue. Under the terms of the deal announced Tuesday, Cambridge, Massachusetts-based Voyager will use its technology to discover capsids that Novartis could potentially use to address three CNS targets. These targets were not disclosed. The deal also gives Novartis the option to acquire capsids for two additional targets agreed to in the future by the partners. In addition to exercise and option fees on these targets, Voyager can earn up to $1.5 billion in milestone payments, as well as royalties on the sale of any commercialized product using the licensed capsids.
The Novartis agreement is similar to Voyager’s deal with Pfizer last OctoberThe deal comes with an upfront payment of $30 million to discover the capsids that can bring two unspecified gene therapies into the central nervous system and heart tissue. The deal follows reports of Voyager’s technical tests on monkeys. The results showed that the capsids discovered by TRACER were better at penetrating the protective blood-brain barrier and transferring their genetic cargo to cells in the brain and spinal cord than traditional capsids. Similar encouraging data have been reported for Voyager capsids against myocardial tissue.
Voyager’s move to form a capsid discovery alliance with Big Pharma is part of a strategic shift that follows several missteps in its gene therapy research.Over the past two years, AbbVie and Neurocrine Biosciences both end gene therapy alliance with VoyagerThe company still has a pipeline of internally developed gene therapies, unlike those covered by its partners, but Voyager sees TRACER as a way to generate new business.
“Our collaboration with Novartis expands the range of therapeutic programs that can deploy our proprietary capsids and highlights the further refinement of our TRACER platform in our novel capsid library expansion and initial TRACER-derived capsids to enhance future business Potential for Growth Opportunities Voyager Chief Commercial Officer Allen Nunnally said in a prepared statement.
There are other companies that are pursuing a similar strategy. Capsida Biotherapeutics launched last May; its technology discovers capsids for its internal gene therapy pipeline and partners including AbbVie. at the same time, Dyno Therapeutics focuses on capsid discovery for other companies. Its partners include Roche, Astellas, Sarepta Therapeutics and Novartis. Novartis’ new partnership with Voyager doesn’t overlap with its Dyno agreement, as earlier alliances specifically covered eye diseases.
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