Novartis and Voyager Therapeutics announce partnership to develop gene therapies for neurological disorders

In a promising step for next-generation gene therapy for neurological diseases, Novartis and Voyager Therapeutics have announced a licence option agreement.

The agreement builds on previous successes from Novartis in using emerging technologies to develop first-in-class gene therapies for neurological disorders.

Novartis is a leading global medicines company, and under the terms of the agreement, will have the right to evaluate novel capsids from Voyager’s TRACER (Tropism Redirection of AAV by Cell type-specific Expression of RNA) platform.

Voyager Therapeutics is a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies. Data suggest that AAV capsids from Voyager’s TRACER platform may show broad and improved distribution to neurons in the cortex and deeper brain regions.

“The challenge of biodistribution of AAV gene therapies to target cell types in the brain is long-standing, but we refuse to believe it is insurmountable,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research (NIBR). “We see the potential of Voyager’s innovative platform to imagine new types of therapeutics for life-threatening CNS diseases.”

Evidence suggests that capsids from the TRACER platform may help deliver therapies to previously intractable regions of the brain. This may help researchers overcome barriers that have hindered the development of gene therapies for neurological diseases for years.

“We have a robust pipeline of gene therapy programs to target diseases of the central nervous system – a number of them requiring enhanced delivery vectors that reach cells in multiple brain regions, which has not been possible with technologies used to date,” commented Bob Baloh, Head of Neuroscience at NIBR. “We look forward to evaluating these capsids from Voyager’s TRACER platform, which hopefully will enable us to bring new therapeutic options to patients with devastating neurological conditions.”

Novartis is a leader in gene therapy and aims to develop treatments for diseases of the central nervous system and for neurological diseases that manifest in deep brain regions where commonly used treatment paths have not been shown to effectively target and penetrate.