Roche and Ascidian Therapeutics have partnered to develop RNA exon editing therapies for neurological diseases, with the deal worth over $1.8bn.
The research collaboration and licensing agreement gives Roche exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed neurological targets.
Ascidian will conduct discovery and certain preclinical activities in collaboration with Roche, which will be responsible for further preclinical work as well as clinical development, manufacturing and commercialisation.
In exchange, Ascidian will receive an initial payment of $42m and will be eligible to receive up to $1.8bn in research, clinical and commercial milestone payments, plus royalties on global commercial sales.
More than three billion people worldwide were living with a neurological condition in 2021, according to a new major study released earlier this year by The Lancet Neurology.
Among the top ten neurological conditions contributing to loss of health in the same year were stroke, neonatal encephalopathy, dementia, diabetic neuropathy, epilepsy and nervous system cancers.
Ascidian said its technology allows for the therapeutic targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. The approach is designed to provide the durability of gene therapy and reduce the risks associated with direct DNA editing and gene replacement.
James Sabry, global head of pharma partnering at Roche, said: “Our partnership with Ascidian is an opportunity to harness advanced RNA exon editing technology, which has the potential to deliver transformative one-time therapeutics by editing multiple whole exons at the RNA level with a single treatment.”
Also commenting on the alliance, Michael Ehlers, Ascidian’s president and chief executive officer, said: “The potential of treating disease by large-scale exon editing of RNA is vast.
“We look forward to working with the Roche team to develop first-in-class RNA exon editing medicines for multiple neurological diseases, with a mission and passion to relieve suffering and improve lives.”
The partnership comes just one week after Roche entered into a strategic licence agreement for the use of ALZpath’s pTau217 antibody to develop and commercialise a diagnostic blood test for Alzheimer’s disease.