Novartis can access Voyager’s AAV capsids for use in therapies for three central nervous system diseases, with options for two additional targets
Novartis Campus Basel, Switzerland. (Credit: Novartis AG)
Novartis has teamed up with Voyager Therapeutics, a gene therapy company engaged in developing advanced adeno-associated virus (AAV) technologies.
The companies have signed a license agreement under which Novartis will obtain the right to evaluate novel capsids from Voyager’s TRACER capsid discovery platform.
The Swiss drugmaker is allowed to use the capsids for the exclusive development of AAV gene therapies against three neurologic diseases.
In addition, the company will have an option to license capsids for two additional central nervous systems (CNS) disease targets that will be finalised in the future.
Voyager is expected to receive an upfront payment of $54m, in addition, to exercise fees of up to $37.5m for three initial CNS targets, which can be exercised by Novartis within 12 months.
Novartis may select the additional two targets by paying $18m for each target and pay $12.5m in exercise fee for selecting a capsid for each target.
In addition, Voyager is eligible to receive up to $1.5bn in potential milestones for products developed under the collaboration, along with royalties on their net sales.
Novartis Institutes for BioMedical Research (NIBR) president Jay Bradner said: “The challenge of biodistribution of AAV gene therapies to target cell types in the brain is longstanding, but we refuse to believe it is insurmountable.
“We see the potential of Voyager’s innovative platform to imagine new types of therapeutics for life-threatening CNS diseases.”
Novartis is expected to leverage its capabilities and recent knowledge in neurological diseases, where usual vectors fail to target and penetrate the deep brain regions.
The agreement builds on its success in using emerging technologies to develop advanced gene therapies for neurological disorders, said Novartis.
Voyager’s TRACER capsid discovery platform is an RNA-based functional screening platform that facilitates the rapid evolution of AAV-derived capsids.
The capsids are said to have enhanced tropisms and cell- and tissue-specific transduction properties in multiple species, including non-human primates (NHPs).
According to the initial data, the capsids effectively penetrated the blood-brain barrier and gained broad biodistribution and transduction of multiple regions of the brain.
Voyager said that it develops additional AAV9-, and AAV5-derived capsids, and other capsid serotypes to identify novel AAV vectors for specific therapeutic applications.
Voyager chief business officer Allen Nunnally said: “This transaction further validates the potential of the Voyager TRACER capsid discovery platform to broadly enable a next-generation of AAV gene therapies.
“Our collaboration with Novartis expands the array of therapeutic programs in which our proprietary capsids may be deployed and highlights the potential of our TRACER platform to generate future business development opportunities as our novel capsid library expands and initial TRACER-derived capsids are further refined to enhance desirable characteristics.”