Ascidian Therapeutics Partners with Roche for RNA Exon Editing in Neurological Diseases

Ascidian Therapeutics, a Boston-based biotechnology firm, has announced a significant collaboration and licensing agreement with Roche to advance RNA exon editing therapeutics aimed at neurological diseases. This partnership leverages Ascidian's innovative RNA exon editing platform alongside Roche's established capabilities in developing treatments for complex neurological conditions.

Under the terms of the agreement, Ascidian will provide Roche with exclusive rights to its RNA exon editing technology for specific neurological targets that remain undisclosed. The collaboration will see Ascidian engaging in discovery and certain preclinical activities alongside Roche. In turn, Roche will handle additional preclinical work, clinical development, manufacturing, and the commercialization of the resulting therapies.

Financially, the deal is substantial. Ascidian will receive an initial payment of $42 million and stands to gain up to $1.8 billion through various research, clinical, and commercial milestone payments. Additionally, Ascidian will earn royalties on the global commercial sales of any successful products. Furthermore, Ascidian retains the freedom to pursue other neurological targets either independently or with other collaborators.

Michael Ehlers, M.D., Ph.D., President, and CEO of Ascidian Therapeutics, expressed pride in the scientific advancements that led to this partnership. He emphasized the expansive potential of RNA exon editing to treat diseases that current gene editing technologies cannot address. Ehlers stated that the collaboration with Roche could lead to first-in-class RNA exon editing medicines for several neurological diseases, aiming to mitigate suffering and enhance lives.

James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche, highlighted the transformative potential of RNA exon editing technology. He pointed out that this approach could allow for one-time treatments by editing multiple exons at the RNA level, significantly impacting the treatment landscape for neurological disorders.

Ascidian's platform is designed to target large genes and those with high mutational variability while preserving native gene expression patterns. This RNA exon editing approach aims to offer the durability associated with gene therapy while minimizing the risks linked to direct DNA editing and gene replacement.

Ascidian Therapeutics, an ATP company, focuses on redefining disease treatment through RNA rewriting. Their therapies enable precise, post-transcriptional gene editing, producing full-length, functional proteins in appropriate cells and at optimal times. The company is actively engaged in discovery, preclinical, and clinical programs targeting retinal, neurological, neuromuscular, and genetically defined diseases. Ascidian's approach could offer one-dose treatments using RNA exon editors, presenting new therapeutic possibilities for patients and their families.

Earlier this year, Ascidian marked a milestone with U.S. FDA IND clearance for ACDN-01, the first RNA exon editing candidate targeting Stargardt disease and other ABCA4 retinopathies. The company is currently conducting the Phase 1/2 STELLAR clinical trial to assess the safety and efficacy of ACDN-01.

This collaboration with Roche underscores the potential of RNA exon editing technology to revolutionize the treatment of neurological diseases, offering hope for novel, effective therapies that address previously untreatable conditions.