Novartis has already commercialized a gene remedy that targets the central nervous system, however the pharmaceutical large has extra CNS gene therapies in its pipeline and it’s seeking to enhance the best way these therapies attain their targets. Voyager Therapeutics has know-how that finds new methods to successfully ship gene therapies into the mind. Novartis sees promise within the Voyager platform, and it has agreed to fork over $54 million to start a multi-therapy alliance.
Most gene therapies are delivered by adeno-associated viruses. The genetic cargo is carried inside a protein shell referred to as a capsid. Novartis’s first gene remedy, the spinal muscular atrophy therapy Zolgensma, makes use of an AAV vector to cross the blood-brain barrier and enter motor neuron cells the place the capsid releases its genetic payload.
The proprietary know-how of Voyager, named TRACER, discovers new AAV capsids able to delivering the gene remedy to explicit varieties of tissue. In accordance with deal phrases introduced Tuesday, Cambridge, Massachusetts-based Voyager will use its know-how to find capsids that Novartis might use to deal with three CNS targets. These targets weren’t disclosed. The deal additionally offers Novartis the choice to entry capsids for 2 further targets that the companions will agree on sooner or later. Along with train and possibility charges for these targets, Voyager may earn as much as $1.5 billion in milestone funds, plus royalties from gross sales of any commercialized merchandise that use the licensed capsids.
The Novartis pact is much like a deal that Voyager signed with Pfizer final October. That deal paid $30 million up entrance for the invention of capsids able to carrying two unspecified gene therapies into CNS and cardiac tissue. That deal adopted Voyager’s report of checks of its know-how in monkeys. The outcomes confirmed that in comparison with standard capsids, the capsids found by TRACER had been higher at penetrating the protecting blood-brain barrier and transferring their genetic cargo to cells within the mind and spinal twine. Comparable encouraging knowledge had been additionally reported for Voyager capsids that focus on cardiac muscle tissue.
Voyager’s transfer to strike up capsid-discovery alliances with massive pharma is a part of a technique shift that follows a number of stumbles for its gene remedy analysis. Previously two years, AbbVie and Neurocrine Biosciences have each terminated gene remedy alliances with Voyager. The corporate nonetheless has a pipeline of internally developed gene therapies separate from these coated by its partnerships, however Voyager sees TRACER as a strategy to generate new enterprise.
“Our collaboration with Novartis expands the array of therapeutic packages by which our proprietary capsids could also be deployed and highlights the potential of our TRACER platform to generate future enterprise growth alternatives as our novel capsid library expands and preliminary TRACER-derived capsids are additional refined to reinforce fascinating traits,” Voyager Chief Enterprise Officer Allen Nunnally stated in a ready assertion.
There are different corporations pursuing comparable methods. Capsida Biotherapeutics launched final Could; its know-how discovers capsids for its inside gene remedy pipeline in addition to for companions that embrace AbbVie. In the meantime, Dyno Therapeutics focuses completely on discovering capsids for different corporations. Its companions embrace Roche, Astellas, Sarepta Therapeutics, and Novartis. Novartis’s new partnership with Voyager doesn’t overlap with its Dyno pact, as that earlier alliance particularly covers eye problems.
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