Roche and Ascidian Therapeutics Collaborate on Treatments for Neurological Diseases

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The research collaboration and licensing agreement will focus on the discovery and development of RNA exon editing therapeutics.

Ascidian Therapeutics, a biotechnology company developing treatments by rewriting RNA, announced on June 18, 2024 that it has signed a licensing agreement with Roche to collaborate on the discovery and development of RNA exon editing therapeutics to treat neurological diseases (1). The collaboration will combine RNA exon editors with Roche’s next-generation central nervous system (CNS) delivery capabilities. Ascidian enables precise post-transcriptional editing of genes by editing exons at the RNA level.

As part of the agreement, Ascidian will receive an initial payment of $42 million with up to $1.8 billion in research, clinical, and commercial milestone payments, including commercial royalties. The partnership will allow Ascidian to create internal and collaborative programs within the neurology area. Roche will receive exclusive, target-specific rights to Ascidian’s RNA exon editing technology and will perform certain preclinical activities. Roche will also be responsible for further clinical development, manufacturing, and commercialization.

“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,” Michael Ehlers, MD, PhD, president and chief executive officer of Ascidian Therapeutics, said in a press release. “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,” James Sabry, MD, PhD, global head of Pharma Partnering at Roche, said in the release.

In May 2024, Ascidian presented preclinical data that enabled the clearance of an investigational new drug (IND) application through Phase I/II testing for its RNA exon editor, ACDN-01, at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore, Md. FDA granted ACDN-01 Rare Pediatric Disease Designation as well as Fast Track Designation.

“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,” said Ehlers in a press release (2). “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. Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing Therapeutics Targeting Neurological Diseases. Press Release. June 18, 2024.
  2. Ascidian Therapeutics. 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. Press Release. April 25, 2024.