Voyager has partnered with Novartis for the latter to use the gene therapy company’s capsid discovery platform to create new treatments for three undisclosed CNS targets.
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Voyager Therapeutics has partnered with Novartis in a potentially $1.7 billion deal. The collaboration will enable Novartis to use the gene therapy company’s capsid discovery platform to create new treatments for three undisclosed CNS targets.
Voyager’s RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform is a broadly applicable, functional screening platform that enables rapid in vivo evolution of AAV9- and AAV5-delivered capsids with enhanced tropisms and cell- and tissue-specific transduction properties in different species.
Multiple studies found that the capsids may have the ability to transduce cardiac muscle and de-target the dorsal root ganglia. Voyager is exploring various capsid campaigns from AAV9, AAV5 and other serotypes to discover vectors that may be optimized for treatment.
Under the terms of the deal, Novartis will pay Voyager $54 million upfront to access its platform and then might add as much as $37.5 million in exercise fees for options for three initial CNS targets. Novartis has 12 months from signing the agreement to exercise its options.
In addition to the said amounts, Novartis will pay $18 million for each target and another $12.5 million as an exercise fee to choose a capsid, should it decide to avail its option to add up to two more targets in the future. Voyager stands to gain as much as $1.5 billion more in development, regulatory and commercial milestones, plus mid-to high-single-digit tiered royalties depending on net sales of capsid-based Novartis products.
The targets Novartis will focus on are different from those already in Voyager’s internal and partnered pipelines. Voyager will maintain its global rights to the TRACER platform and the capsids generated through it for use with other disease targets. These will be subject to Voyager’s agreement in October 2021 with Pfizer, which gives the latter access to the TRACER platform to use capsids for one cardiovascular and one neurological target.
“Overcoming the substantial toxicity risk frequently observed with high doses of AAV-based gene therapies remains a critical challenge to realizing the full, curative potential of this breakthrough modality. Voyager’s growing and maturing library of proprietary TRACER-derived capsids have demonstrated markedly enhanced expression in non-human primates with more precise targeting of desired tissue and cell types, creating the potential for superior delivery and fewer off-target risks than conventional AAV,” said Glenn Pierce, MD, Ph.D., the interim chief scientific officer of Voyager, in a statement.
In October 2021, Pfizer gave Voyager $30 million upfront plus a promise of potential milestone payments of up to $600 million plus royalties to use the latter’s TRACER technology to pursue its treatment discovery and development of two undisclosed transgenes linked to the central nervous system and cardiac tropisms. Similar to the Novartis deal, Pfizer also has 12 months to exercise its two options in the amount of $20 million. Voyager retains worldwide rights to all licensed capsids related to its technology.
