Novartis signs gene therapy deal with Voyager for US$100 million upfront

Voyager Therapeutics said on Tuesday (Jan 2) Swiss-based drugmaker Novartis would pay US$100 million upfront as part of a licensing deal to develop gene therapy candidates for genetic disorders.

Voyager, whose shares jumped more than 30 per cent in premarket trading, would be eligible to receive up to US$1.2 billion on achieving certain milestones.

The gene therapy developer would provide Novartis 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.

Voyager would advance the pre-clinical development and Novartis would be responsible for all clinical studies and commercialisation for the HD candidate.

Novartis would also gain access to Voyager’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.

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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.

Separately, Voyager is developing an investigational gene therapy to treat a neurological disease, amyotrophic lateral sclerosis, and an antibody for the Alzheimer’s disease. REUTERS