Roche, Ascidian to develop gene therapies for neurological diseases

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Roche has announced a research collaboration and licensing agreement with biotech firm Ascidian Therapeutics to develop gene therapies for neurological diseases.

According to the terms of their agreement, Roche will pay Ascidian $42 million upfront for exclusive, target-specific rights to use Ascidian's RNA exon-editing technology to develop therapeutics for undisclosed neurological diseases, the firms said in a statement. Furthermore, Roche will pay Ascidian up to $1.8 billion in total as research, clinical, and commercial milestones are reached. Ascidian is also eligible to receive royalties on commercial sales worldwide for any therapies that are developed under the collaboration.

Under their arrangement, Ascidian will be responsible for conducting discovery and certain preclinical activities in collaboration with Roche, and Roche will be responsible for certain other preclinical activities, as well as further clinical development, manufacturing, and commercialization, Ascidian said. While Roche will have exclusive rights to the technology for the targeted diseases, Ascidian can pursue other disease targets outside of its agreement with Roche.

Boston-based Ascidian's exon-editing technology is designed to correct the RNA produced by damaged exons, the regions of DNA containing the blueprints to make proteins. Correcting the RNA produced by damaged exons greatly mitigates the risks associated with direct DNA editing and gene replacement, such as off-target edits, Ascidian said.

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