Roche partners with Ascidian for RNA exon editing

Roche has entered into a research collaboration and licensing agreement with Ascidian Therapeutics, a biotech startup based in Boston. The partnership focuses on developing RNA exon editing therapeutics aimed at treating neurological diseases. Ascidian's innovative RNA exon editing platform is designed to replace several contiguous exons rather than just single bases, which opens up new possibilities for RNA medicine. This platform offers a more versatile approach to treating diseases that current gene editing technologies cannot address.

Through this deal, Roche gains exclusive rights to apply Ascidian’s RNA exon editing technology to specific targets related to neurological conditions, which have not been disclosed. In return, Ascidian will receive an upfront payment of $42 million. Additionally, the startup stands to earn up to $1.8 billion in milestone payments, alongside royalties from global commercial sales.

Earlier this year, in January, the U.S. Food and Drug Administration (FDA) approved Ascidian's Investigational New Drug (IND) application and granted Fast Track designation for the company's RNA exon editor intended for retinal diseases. This approval is notable as it marks the first instance of an RNA editor being cleared to enter clinical trials in the United States. Ascidian's premier treatment, ACDN-01, is currently the only therapeutic in clinical stages that targets the genetic basis of Stargardt disease, the most prevalent form of inherited macular degeneration.

This agreement with Ascidian is Roche's second significant RNA-related deal this year. Earlier in January, Roche announced a potential $1 billion collaboration with Remix Therapeutics. This collaboration aims to discover and develop small molecule therapeutics that influence RNA processing using Remix’s REMaster drug discovery platform.

In summary, Roche is significantly expanding its involvement in RNA-based therapeutics through strategic collaborations. The agreement with Ascidian Therapeutics offers promising advancements for treating neurological diseases through innovative RNA exon editing technology. Meanwhile, the collaboration with Remix Therapeutics further underscores Roche’s commitment to pioneering RNA medicine, aiming to transform the treatment landscape for various genetic conditions.