Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, has entered a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.

Ascidian’s RNA exon editing platform is designed to advance the therapeutic possibilities of RNA medicine and treat diseases not addressed by today’s gene editing technologies. The company designs and develops RNA exon editing therapeutics that edit RNA exons at the kilobase scale.

Under the agreement, Ascidian will provide 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, and Roche will be responsible for certain preclinical activities, and further 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.

“Roche is known and respected worldwide for their expertise in complex neurological diseases, and I am proud of the scientific rigor and quality of the work done at Ascidian that has led to this partnership,” said Michael Ehlers, M.D., Ph.D., President and CEO of Ascidian Therapeutics. “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.”

“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,” added James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche.

Ascidian’s platform enables targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. By rewriting RNA, Ascidian’s exon editing technology is designed to provide the durability of gene therapy, while sharply reducing risks associated with direct DNA editing and gene replacement.

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