RARE Daily

Voyager Enters License Option Agreement with Novartis for Target-Specific Access to Its AAV Capsids

March 8, 2022

Voyager Therapeutics entered an option exercise agreement with Novartis to license novel AAV capsids generated from Voyager’s RNA-driven TRACER capsid discovery platform for potential use with three undisclosed CNS targets and options to access capsids for two additional targets to be agreed on in the future.

Photo: Allen Nunnally, chief business officer of Voyager

Voyager will receive $54 million upfront and is entitled to receive up to $37.5 million in exercise fees for options for three initial CNS targets, exercisable by Novartis within 12 months of signing. In addition, Novartis may elect to evaluate capsids for up to two additional targets to be agreed on in the future, subject to their availability, for $18 million upon selection of each target, and a $12.5 million exercise fee for selection of a capsid for each target. Voyager is also eligible to earn up to $1.5 billion in potential development, regulatory, and commercial milestones for products utilizing Voyager licensed capsids, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.

“This transaction further validates the potential of the Voyager TRACER capsid discovery platform to broadly enable a next generation of AAV gene therapies,” said Allen Nunnally, chief business officer of Voyager. “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.”

After recent setbacks to its clinical programs, Voyager has turned back to drug discovery, focusing on its TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform as a broadly applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV9- and AAV5-derived capsids with enhanced tropisms and cell- and tissue-specific transduction properties in multiple species, including non-human primates (NHPs).

Under the terms of the agreement, Novartis has the right to evaluate novel capsids from Voyager’s TRACER platform and to exercise options to license capsids for exclusive use with specific targets in Novartis’ development of AAV gene therapies. The targets for which Novartis receives rights under the agreement are distinct from those in Voyager’s internal and partnered pipeline, and Voyager retains global rights to its TRACER discovery platform as well as all capsids arising from it for use with other targets, subject to Voyager’s obligations under its agreement with Pfizer announced in October 2021 for access to TRACER capsids for use with one neurologic and one cardiovascular target.

An initial set of proprietary AAV capsids derived from Voyager’s TRACER platform have demonstrated superior blood-brain barrier penetration, increased transduction in the brain and spinal cord, enhanced cardiac muscle tropism, and increased transgene expression in target tissues compared to conventional AAV capsids when dosed intravenously in non-human primates (NHPs).

Subsequent TRACER screening campaigns have identified a new class of AAV9 variants selective for glial cells, which may enable more precise targeting of CNS diseases affecting non-neuronal cells, as well as AAV5 capsid variants with enhanced brain and spinal cord tropism, compared to conventional AAV9 and AAV5. Voyager is performing further screening with its TRACER capsid discovery platform to identify additional proprietary AAV9- and AAV5-derived capsids targeting multiple tissue and cell types, as well as further refining identified capsids to enhance desirable characteristics for use in gene therapies to treat a broad range of diseases.

Author: Rare Daily Staff

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