Roche Inks Potential $1.8B Deal With Ascidian to Develop RNA Exon Therapies

Pictured: Exterior of Roche's facility in Boulogne

Pictured: Exterior of Roche’s facility in Boulogne

Ascidian Therapeutics will receive $42 million upfront from Roche, and up to $1.8 billion in milestone payments and royalties, to discover RNA exon editing candidates for neurological diseases.

Pictured: Exterior of Roche’s facility in Boulogne-Billancourt, France, iStock/HJBC

Ascidian Therapeutics announced Tuesday that it has signed a potential $1.8 billion partnership with Roche, which will pay $42 million upfront to develop RNA exon editing therapeutics targeting neurological diseases.

Ascidian, which was named to BioSpace’s NextGen Class of 2024, will provide Roche with target-specific rights to its RNA exon editing technology for an undisclosed number of neurological targets. In addition to the upfront cash, Ascidian is eligible receive up to $1.8 billion in research, clinical and commercial milestones, as well as royalties on sales globally.

Under the agreement, the Boston-based biotech will be responsible for conducting discovery and preclinical activities in collaboration with Roche. The Swiss pharma, for its part, will be responsible for other preclinical work on the assets as well as future clinical development, manufacturing and commercialization. Ascidian is also free to develop programs for other neurological targets internally or with 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,” Ascidian CEO Michael Ehlers said in a statement. “The potential of treating disease by large-scale exon editing of RNA is vast.”

The biotech’s approach involves using a platform that targets large genes and genes with high mutational variance while keeping the native gene expression. Ascidian’s technology is designed to make exon swaps in RNA transcripts that encode proteins. The company is developing other assets and received IND clearance from the FDA in January 2024 for its asset ACDN-01 to treat the genetic eye condition Stargardt disease targeting the genetic cause of the condition.

“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,” James Sabry, the global head of pharma partnering at Roche, said in a statement.

Roche has been on a deal-making spree over the past year. In December 2023, it spent $2.7 billion purchasing Carmot Therapeutics and gaining access to its GLP-1 weight loss assets. In January 2024, Roche inked a possible $1 billion deal with China-based MediLink Therapeutics to develop an antibody-drug conjugate in oncology and a potential $2 billion deal with MOMA Therapeutics to find new drugs to target cancer cell growth and survival.

Tyler Patchen is a staff writer at BioSpace. You can reach him at Follow him on LinkedIn.

Tyler Patchen is a staff writer at BioSpace. You can reach him at Follow him on LinkedIn.