US-based biotechnology company Ascidian Therapeutics has partnered with Swiss pharmaceutical firm Roche, for the discovery and development of RNA therapeutics for neurological diseases.
The collaboration will combine Ascidian’s RNA exon editing platform with Roche’s advanced CNS delivery capabilities to develop novel RNA therapeutics targeting neurological diseases.
Under the terms of the agreement, Roche will obtain exclusive, target-specific rights to Ascidian’s RNA exon editing technology.
Ascidian will be responsible for the discovery and certain preclinical activities together with Roche, which will lead the further clinical development, manufacturing, and commercialisation.
The US drugmaker will receive an initial payment of $42m and is eligible to receive up to $1.8bn in research, clinical, and commercial milestone payments, along with royalties on global sales.
It can develop programs against other neurological targets alone or in collaboration with others.
Roche pharma partnering global head James Sabry 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.”
Ascidian has developed an RNA exon editing platform to advance the therapeutic possibilities of RNA medicine and treat diseases that are not addressed by current gene editing technologies.
The company designs and develops RNA therapeutics that edit RNA exons at a kilobase scale.
Its platform enables the targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels.
The exon editing technology rewrites RNA to provide the durability of gene therapy, while sharply reducing risks associated with direct DNA editing and gene replacement.
Ascidian Therapeutics president and CEO Michael Ehlers said: “Roche is known and respected worldwide for their expertise in complex neurological diseases, and I am proud of the scientific rigour and quality of the work done at Ascidian that has led to this partnership.
“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.”
