Novartis Inks Potential $1.3B Gene Therapy Deal with Voyager to Target Huntington’s Disease and Spinal Muscular Atrophy

LEXINGTON, Mass. — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA). 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 HD.

“We are thrilled to expand our existing relationship with Novartis, a global leader in the gene therapy field,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “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. In addition, the consideration Voyager will receive from this collaboration will strengthen our balance sheet and extend our runway into mid-2026.”

“We look forward to broadening our work with Voyager to help bring forward novel, high-impact gene therapies with the potential to improve the lives of patients affected by severe neurologic conditions,” said Fiona Marshall, President of Biomedical Research at Novartis. “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.”

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.

 

Collaboration Details and Financial Terms
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.

Chestnut Partners served as exclusive financial advisor to Voyager on this transaction.

 

About the TRACER™ Capsid Discovery Platform
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 (NHPs). 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. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

 

About Voyager Therapeutics
Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc.

 

Contacts

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Media
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Peg Rusconi
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