Roche and Ascidian Therapeutics partner to develop RNA editing therapeutics

By Clara Rodriguez Fernandez

- Last updated on GMT

Image: Getty/ Natali_Mis
Image: Getty/ Natali_Mis

Related tags Rna Roche Gene editing

With Roche as a partner, Ascidian Therapeutics aims to treat conditions that currently cannot be addressed by existing gene editing technology

Ascidian Therapeutics and Roche have started a research collaboration and licensing agreement to develop RNA exon editing therapeutics. As part of the deal, Ascidian Therapeutics will receive a $42 million initial payment from Roche and up to $1.8 billion in milestone payments, as well as royalties on worldwide sales. Ascidian will conduct discovery and early preclinical activities, while Roche will be responsible for further preclinical and clinical development, manufacturing, and commercialization.

The treatments developed under the partnership will target a series of difficult-to-treat neurological diseases, focusing on conditions affecting the central nervous system. "Roche is known and respected worldwide for their expertise in complex neurological diseases," commented Michael Ehlers, President and Chief Executive Officer of Ascidian Therapeutics.

Potential of RNA exon editing

Ascidian Therapeutics is a biotechnology company developing therapeutics that edit RNA exons, which are sections of an RNA molecule that get translated into proteins. These RNA medicines have the potential to treat diseases that are not addressed by existing gene editing technologies.

"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," said Ehlers.

Addressing genetic mutations

Ascidian’s exon editing platform is designed to address the effects of genetic mutations by editing RNA, which provides the same durability as gene therapy but without the risks associated with editing DNA directly.

"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," said James Sabry, Global Head of Pharma Partnering at Roche.

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