Novartis has already commercialized a gene therapy that targets the central nervous system, but the pharmaceutical giant has more CNS gene therapies in its pipeline and it’s looking to improve the way these therapies reach their targets. Voyager Therapeutics has technology that finds new ways to effectively deliver gene therapies into the brain. Novartis sees promise in the Voyager platform, and it has agreed to fork over $54 million to begin a multi-therapy alliance.
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Most gene therapies are delivered by adeno-associated viruses. The genetic cargo is carried inside a protein shell called a capsid. Novartis’s first gene therapy, the 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.
The proprietary technology of Voyager, named TRACER, discovers new AAV capsids capable of delivering the gene therapy to particular types of tissue. According to deal terms announced Tuesday, Cambridge, Massachusetts-based Voyager will use its technology to discover capsids that Novartis may use to address three CNS targets. Those targets were not disclosed. The deal also gives Novartis the option to access capsids for two additional targets that the partners will agree on in the future. In addition to exercise and option fees for those targets, Voyager could earn up to $1.5 billion in milestone payments, plus royalties from sales of any commercialized products that use the licensed capsids.
The Novartis pact is similar to a deal that Voyager signed with Pfizer last October. That deal paid $30 million up front for the discovery of capsids capable of carrying two unspecified gene therapies into CNS and cardiac tissue. That deal followed Voyager’s report of tests of its technology in monkeys. The results showed that compared to conventional capsids, 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. Similar encouraging data were also reported for Voyager capsids that target cardiac muscle tissue.
Voyager’s move to strike up capsid-discovery alliances with big pharma is part of a strategy shift that follows several stumbles for its gene therapy research. In the past two years, AbbVie and Neurocrine Biosciences have both terminated gene therapy alliances with Voyager. The company still has a pipeline of internally developed gene therapies separate from those covered by its partnerships, but Voyager sees TRACER as a way to generate new business.
“Our collaboration with Novartis expands the array of therapeutic programs in which our proprietary capsids may be deployed and highlights the potential of our TRACER platform to generate future business development opportunities as our novel capsid library expands and initial TRACER-derived capsids are further refined to enhance desirable characteristics,” Voyager Chief Business Officer Allen Nunnally said in a prepared statement.
There are other companies pursuing similar strategies. Capsida Biotherapeutics launched last May; its technology discovers capsids for its internal gene therapy pipeline as well as for partners that include AbbVie. Meanwhile, Dyno Therapeutics focuses exclusively on discovering capsids for other companies. Its partners include Roche, Astellas, Sarepta Therapeutics, and Novartis. Novartis’s new partnership with Voyager does not overlap with its Dyno pact, as that earlier alliance specifically covers eye disorders.
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