SWITZERLAND – Voyager Therapeutics has secured a license option agreement in a potentially US$1.8 billion deal with Novartis.

Novartis will be able to use the gene therapy company’s capsid discovery platform to develop new treatments for three unnamed CNS targets as a result of the collaboration.

The TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform from Voyager is a broadly applicable, functional screening platform that enables rapid in vivo evolution of Adeno Associated Virus 9- (AAV9) and AAV5-delivered capsids with enhanced tropisms and cell- and tissue-specific transduction properties in various species.

Voyager’s TRACER technology platform adeno-associated virus (AAV) capsids demonstrated superior blood-brain barrier penetration, improved cardiac muscle tropism, and increased transgene expression in target tissues.

To put it another way, the capsids appear to target cells and tissues more precisely and at lower doses. There appear to be fewer off-target issues than with traditional AAVs.

Voyager is investigating various capsid campaigns from Adeno Associated Virus 9 (AAV9), AAV5, and other serotypes in order to identify vectors that could be optimized for treatment.

In a US$1.8 billion deal, Voyager Therapeutics will engineer capsids to deliver gene therapies into patients’ cells for Novartis.

According to the terms of the agreement, Novartis will pay Voyager US$54 million upfront to gain access to its platform, plus up to US$37.5 million in exercise fees for three initial CNS targets.

Novartis has 12 months from the date the agreement was signed to exercise its options.

In addition to the aforementioned amounts, Novartis will pay US$18 million for each target and another US$12.5 million as an exercise fee to select a capsid if it chooses to exercise its option to add up to two more targets in the future.

Voyager could earn up to US$1.5 billion more in development, regulatory, and commercial milestones, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis capsid-based products.

The targets Novartis will pursue are distinct from those already in Voyager’s internal and collaborative pipelines.

Voyager will retain global rights to the TRACER platform and the capsids generated by it for future use with other disease targets.

These will be subject to Voyager’s agreement with Pfizer in October 2021, which grants the latter access to the TRACER platform in order to use capsids for one cardiovascular and one neurological target.

In October 2021, Pfizer paid Voyager US$30 million up front, with the promise of up to US$600 million in milestone payments and royalties if the latter used its TRACER technology to pursue treatment discovery and development of two undisclosed transgenes linked to central nervous system and cardiac tropisms.

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