Ascidian Therapeutics, Roche Enter RNA Exon Editing Therapeutics Collaboration


Deal will target neurological diseases, featuring an initial payment of $42 million for Ascidian and up to $1.8 billion in milestone payments/royalties.

Image Credit: Adobe Stock Images/

Image Credit: Adobe Stock Images/

Ascidian Therapeutics, a biotech company, has entered a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon-editing therapeutics that target neurological diseases.1

Ascidian—whose efforts revolve around rewriting RNA—has an RNA exon editing platform that is intended to further the benefits of RNA medicine by tackling diseases that are not necessarily treated by current gene editing tech. In essence, the company works to create RNA exon editing therapeutics that edit RNA exons at the kilobase scale.

Per the terms of the agreement, Ascidian is expected to grant Roche with exclusive, target-specific rights to its aforementioned RNA exon editing technology for neurological targets that are yet to be revealed at this time. Together, the biotech company will also be responsible for leading any discovery efforts along with any preclinical with Roche, while also expanding on additional clinical development efforts, the manufacturing process, and commercialization.

The deal also states that Ascidian will initially be awarded $42 million, with the potential for up to $1.8 billion in not only international commercial sales royalties, but also research, clinical, and commercial milestone payments. Ascidian is able to develop programs that target other neurological targets on their own or with other parties.

"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," commented Michael Ehlers, MD, PhD, president and CEO, Ascidian Therapeutics. "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, with a mission and passion to relieve suffering and improve lives."

"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, MD, PhD, Roche’s global head of pharma partnering.

The company had shared preclinical data that enabled IND clearance for Phase I/II testing for its main RNA exon editing therapy, ACDN-01, at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore last month. This therapy was also granted Rare Pediatric Disease Designation for by the FDA.2

“ACDN-01 is the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease, and we look forward to sharing the preclinical data that led to its IND clearance, along with the plans for the first-in-human trial,” added Ehlers. “ACDN-01 is designed to overcome challenges that have long kept Stargardt disease out of reach and to provide persistent therapeutic benefit with one dose. Our progress with ACDN-01 speaks to its therapeutic potential in Stargardt disease and the broader promise of our RNA exon editing platform to significantly expand the possibilities of RNA medicines.”


1. Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing Therapeutics Targeting Neurological Diseases. Ascidian Therapeutics. June 18, 2024. Accessed June 19, 2024.

2. Ascidian Therapeutics to Present Preclinical Data for RNA Exon Editor, ACDN-01, Supporting an Open IND for Phase 1/2 Clinical Testing at ASGCT 2024 Annual Meeting. Ascidian Therapeutics. April 25, 2024. Accessed June 19, 2024.

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