January 2, 2024 10:45am

Novartis would pay $100 million upfront as part of a licensing deal to develop gene therapy candidates. The gene therapies developer is also eligible to receive up to $1.2 billion in certain milestone payments, as well as tiered royalties on global net sales of products incorporating its RNA-based screening platform.

VYGR shares jumped 28.31% in morning trading ...

Providing NVS a target-exclusive license to access VYGR's RNA-based screening platform and advance a gene therapy candidate in pre-clinical stage for a genetic disorder known as Huntington's disease (HD).

The Bottom Line: The gene therapy developer would provide NVS access to its RNA-based screening platform — which helps in rapid discovery of experimental gene therapies — and would also be eligible for tiered royalties on global sales of products developed using the platform.

The companies would collaborate to develop a pre-clinical gene therapy candidate for Huntington's disease (HD), an inherited condition that causes nerve cells in parts of the brain to gradually break down and die.

  • VYGR would advance the pre-clinical development and Novartis would be responsible for all clinical studies and commercialization for the HD candidate.

NVS would also gain access to VYGR's platform for discovery and development of potential gene therapies for treating spinal muscular atrophy, a group of rare genetic disorders which affect the nerve cells and cause muscle wasting and weakness.

  • Novartis had previously exercised an option to license novel capsids or gene therapy delivery vehicles generated from Voyager's drug discovery platform as a potential treatment for two undisclosed neurological conditions.

Voyager is ALSO ... developing an investigational gene therapy to treat a neurological disease, amyotrophic lateral sclerosis (ALS), and an antibody for the Alzheimer's disease.