Ascidian Therapeutics and Roche Seal Strategic Research Partnership for Future Medical Advancements

Ascidian Therapeutics, a biotech firm dedicated to treating diseases by modifying RNA, has partnered with Roche in a research collaboration and licensing agreement to discover and develop RNA exon editing therapies for neurological conditions. The editing platform of Ascidian's RNA exon editing platform aims to expand the potential of RNA-based medicine and address diseases that current gene editing technologies cannot! The company tends to focus on creating RNA exon editing therapies that operate at the kilobase scale.

According to the agreement, Ascidian will grant Roche exclusive, target-specific access to its RNA exon editing technology for unspecified neurological targets. Ascidian will handle discovery and some preclinical activities in partnership with Roche, while Roche will take on additional preclinical tasks, as well as clinical development, manufacturing, and commercialization.

Ascidian will receive an initial payment of $42 million and is eligible to receive up to $1.8 billion in research, clinical, and commercial milestone payments, as well as royalties on commercial sales worldwide. Based on the terms of the agreement, Ascidian is free to develop programs against other neurological targets internally or with other collaborators.

 Michael Ehlers, M.D., Ph.D., President and CEO of Ascidian Therapeutics 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.

He further said that “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.”

James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche added, "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's platform allows for the targeting of extensive genes and those with significant mutational diversity, all while preserving the natural patterns and levels of gene expression. By having a focus towards RNA editing, Ascidian's exon editing technology aims to offer the long-lasting effects of gene therapy, significantly minimizing the risks linked to direct DNA modification and gene replacement.