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Novartis turns to Voyager for nervous system gene therapies

Already a heavyweight in the fields of gene therapy and neuroscience research, Novartis hopes to create more genetic medicines for nervous system disorders through a new deal that provides access to technology developed by Voyager Therapeutics.

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Announced Tuesday, the deal allows Novartis to initially license three of Voyager's novel capsids, which are the outer coatings that protect gene therapies until they're delivered to the right cells. Later, Novartis may license up to two additional capsids. Deal terms hold that Novartis would have exclusive access to these viral shells, but only for specified targets in the central nervous system, which the companies have yet to disclose.

Novartis will pay Voyager $54 million upfront and as much as $37.5 million in licensing fees related to the three initial targets. To further expand the deal, Novartis could deploy Voyager's technology to two more targets for a price of $18 million per target plus a $12.5 million fee related to the capsid it selects. Voyager could also earn up to $1.5 billion in various milestone payments.

Cellular and genetic medicines have the potential to effectively cure a wide range of diseases. Yet, only in the last decade or so has cutting-edge research translated to marketed products. In late 2017, Roche's Luxturna became the first gene therapy cleared in the U.S. to treat an inherited disease. And less than two years later, Novartis received approval for Zolgensma, a genetic medicine for a rare neurological disorder.

As a result, investment in this space has also soared as large pharmaceutical firms, looking to quickly ramp up their research efforts, partnered with or acquired promising biotechs specialized in cell and gene therapy research. Luxturna and Zolgensma, for instance, were respectively developed by Spark Therapeutics and AveXis before multibillion-dollar deals folded them into Roche and Novartis.

Novartis remains one of the most prolific gene therapy dealmakers. Not long after AveXis, it dropped $150 million to acquire Vedere Bio. Then, in the back half of last year, Novartis snagged Arctos Medical for an undisclosed sum and Gyroscope Therapeutics for $800 million. All three deals revolve around genetic medicines for the eye.

The new agreement with Voyager, meanwhile, focuses on a notoriously difficult area of the body to treat. The central nervous system is protected by a wall of cells; and while this so-called blood-brain barrier preserves the system, it is also extremely effective at keeping out foreign substances, including potential medicines.

Novartis is betting that Voyager's capsids can be used to design gene therapies made with adeno-associated viruses, or AAVs, that can overcome some of these delivery and distribution problems.

"The challenge of biodistribution of AAV gene therapies to target cell types in the brain is longstanding, but we refuse to believe it is insurmountable," Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in a statement Tuesday. "We see the potential of Voyager’s innovative platform to imagine new types of therapeutics for life-threatening [central nervous system] diseases."

Novartis isn't alone, either. Pfizer recently entered a similar-structured deal with Voyager, agreeing to pay $30 million upfront for the option to license two capsids for gene therapies targeting unspecified neurological and cardiovascular diseases.

Other innovative capsid technologies, such as the one developed by Dyno Therapeutics, have attracted attention from large gene therapy developers as well. Since its debut in May 2020, Dyno has inked deals with Novartis, Roche, Astellas and Sarepta Therapeutics.

Published March 8, 2022

Jacob BellSenior Reporter

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