RARE Daily

Voyager Enters Capsid License Collaboration with Novartis to Advance Novel Gene Therapies

January 2, 2024

Rare Daily Staff

Voyager Therapeutics entered a strategic collaboration and capsid license agreement with Novartis Pharma to advance potential gene therapies for the rare neurological diseases Huntington’s disease and spinal muscular atrophy.

Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for Huntington’s disease (HD).

Voyager’s TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates.

In preclinical studies, TRACER generated capsids have demonstrated widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia.

Under the terms of the agreement, Novartis has agreed to pay Voyager $100 million of consideration up front, including a $20 million purchase of newly issued equity in Voyager. Voyager is eligible to receive up to $1.2 billion in preclinical, development, regulatory and sales milestones, as well as tiered royalties on global net sales of products incorporating Voyager’s TRACER capsids. Novartis will obtain target-exclusive access to Voyager’s TRACER capsids related to SMA for the duration of the agreement and will be responsible for all development and commercialization. Novartis will also receive worldwide rights to Voyager’s AAV gene therapy for HD, leveraging Voyager’s TRACER capsids and proprietary payloads. Voyager will be responsible for preclinical advancement and Novartis will be responsible for all clinical development and commercialization for the HD program.

“Combining the proven capabilities of Novartis in gene therapy development and commercialization with Voyager’s next-generation TRACER capsids and payloads could enable the advancement of important new therapies for patients,” said Alfred Sandrock, CEO of Voyager. “In addition, the consideration Voyager will receive from this collaboration will strengthen our balance sheet and extend our runway into mid-2026.”

Novartis previously exercised options to license novel capsids generated from Voyager’s TRACER capsid discovery platform for use in gene therapy programs against two undisclosed neurological disease targets.

“We believe Voyager’s TRACER capsids hold promise for enabling next-generation gene therapies for diseases of the central nervous system, aligning well with our deep neuroscience expertise and gene therapy leadership at Novartis,” said Fiona Marshall, president of Biomedical Research at Novartis.

Photo: Alfred Sandrock, CEO of Voyager

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