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Roche Partners with RNA Editing Biotech Ascidian

The Swiss pharma will pay $42 million to gain rights to use Ascidian’s RNA exon editing technology for certain neurology drug targets.

Roche has partnered with Ascidian Therapeutics to develop so-called “RNA exon editing” drugs, the latest foray by the Swiss pharmaceutical company to shore up its neurology pipeline.

Under the deal, announced by the companies Tuesday, Roche will pay Ascidian $42 million upfront, and has promised as much as $1.8 billion more if certain development and commercial milestones are hit. The collaboration gives Roche exclusive rights to Ascidian’s editing technology for certain undisclosed neurological disease targets.

Named for a sea creature that can re-engineer its own RNA, Ascidian launched in the fall of 2022 with $50 million from the venture firm ATP. The company aims to treat disease by correcting the RNA produced by damaged exons, the regions of DNA that contain the blueprints to make proteins.

Biotechs like Ascidian have turned their focus to RNA to sidestep some of the problems that can result by editing DNA directly, such as off-target edits.

“There are many, incredible teams doing great work on technology that will be very difficult to translate into a therapy that goes into a patient,” said Dan Rosan, Ascidian’s chief financial and business officer. “We have been totally focused on translating our technology into drugs.”

Earlier this year, Ascidian received a green light from the Food and Drug Administration to begin clinical testing of its first RNA exon editing drug, which it’s testing in Stargardt’s disease. A genetic eye disease, Stargardt’s is the most common form of inherited macular degeneration and eventually results in vision loss.

Ascidian is free to pursue other neurological disease targets outside of its collaboration with Roche, the companies said.

“Patients are waiting with hope and now it’s about delivery,” said Robert Bell, the company’s head of research. “We’re really enthusiastic about the adventure ahead with the Roche team.”

For Roche, the deal is an example of its continued neuroscience focus, even as it and other pharma peers have experienced research setbacks. The company is advancing an Alzheimer’s drug, trotinemab, that’s shown some promise, and in September inked a deal with Ionis Pharmaceuticals for two preclinical RNA medicines.

Yet in February, it cut five neurology programs, as well as a handful of early-stage cancers, as it laid new plans for obesity and autoimmune disease drug development.