Pharma Focus Europe

Ascidian Therapeutics Collaborates with Roche on RNA Exon Editing Treatments for Neurological Disorders

Wednesday, June 19, 2024

Ascidian Therapeutics, a company that specializes in RNA-based therapies, has entered into a research collaboration and licensing agreement with Roche. This collaboration aims to develop RNA exon editing therapeutics to address neurological diseases.

Ascidian's RNA exon editing platform offers a novel approach to RNA medicine, enabling precise editing of RNA exons at the kilobase level. According to the terms of the agreement, Ascidian will provide Roche with exclusive rights to this technology for certain undisclosed neurological targets. Ascidian will lead the discovery phase and specific preclinical activities in collaboration with Roche, which will be responsible for additional preclinical activities, as well as clinical development, manufacturing, and commercialization. Ascidian will receive an initial payment of $42 million and could earn up to $1.8 billion in research, clinical, and commercial milestones, in addition to royalties on worldwide sales. The arrangement also permits Ascidian to independently develop other neurological targets or collaborate with different partners.

Ascidian Therapeutics, expressed enthusiasm about the partnership, noting the potential of RNA exon editing to transform treatments for complex neurological conditions. He is eager to collaborate with Roche to create pioneering RNA exon editing therapies that could significantly improve patients' lives.

James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche, highlighted the potential of Ascidian's technology to deliver innovative, one-time treatments that can edit multiple RNA exons simultaneously.

Ascidian's technology allows for the targeting of large genes and those with significant genetic variability while maintaining natural gene expression patterns. This approach provides the long-term benefits of gene therapy while reducing the risks associated with direct DNA editing and gene replacement.



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