Roche has signed a research collaboration and licensing agreement with Boston-based biotech startup Ascidian Therapeutics aimed at the discovery and development of RNA exon editing therapeutics targeting neurological diseases.
Ascidian's RNA exon editing platform is designed to expand the therapeutic possibilities of RNA medicine by replacing multiple contiguous exons — not just single bases — providing a more versatile RNA therapeutic approach and treating diseases not addressed by today’s gene editing technologies.
The deal gives Roche exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed neurological targets. In exchange, Ascidian will receive an initial payment of $42 million and is eligible to receive up to $1.8 billion in milestone payments, as well as royalties on commercial sales worldwide.
Back in January, the FDA greenlighted Ascidian's IND application and granted Fast Track designation for its RNA exon editor in retinal diseases — marking the first time an RNA editor has been cleared in the U.S. to enter the clinic. Ascidian's lead treatment, ACDN-01, is the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease, the most common form of inherited macular degeneration.
This is Roche's second RNA deal of the year. The drugmaker inked a potential $1 billion collaboration with Remix Therapeutics back in January for the discovery and development of small molecule therapeutics that modulate RNA processing using Remix’s REMaster drug discovery platform.