The Cambridge, Massachusetts-based company said that, under the agreement, Novartis receives target-specific access to Voyager’s novel Tracer adeno-associated virus (AAV) capsids for potential use with three central nervous system (CNS) targets along with options to access capsids for two additional targets, to be decided on in the future.
Voyager will receive an upfront payment of $54m and will be eligible for up to $1.7bn in option exercise fees and milestone payments, along with sales-based royalties.
The targets for which Novartis receives rights under the agreement are distinct from those in Voyager’s internal and partnered pipeline.
Voyager retains global rights to its Tropism Redirection of AAV by Cell-type-specific Expression of RNA (Tracer) screening technology as well as all capsids arising from it for use with other targets, subject to the company’s obligations under its agreement with Pfizer, announced in October 2021, and worth $630m.
That deal gave Pfizer access to Tracer capsids for use with one neurologic and one cardiovascular target as part of the pharma giant’s efforts to develop, manufacture, and commercialize gene therapies, utilizing two undisclosed transgenes to treat such diseases.
“Overcoming the substantial toxicity risk frequently observed with high doses of AAV-based gene therapies remains a critical challenge to realizing the full, curative potential of this breakthrough modality,” said Glenn Pierce, interim chief scientific officer, Voyager.
“Voyager’s growing and maturing library of proprietary Tracer-derived capsids have demonstrated markedly enhanced expression in non-human primates with more precise targeting of desired tissue and cell types, creating the potential for superior delivery and fewer off-target risks than conventional AAV," he added.
The company claims an initial set of AAV capsids derived from its Tracer platform 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).