RNA Rewriting to Treat Neurological Disease Focus of Ascidian, Roche Collaboration

Ascidian Therapeutics, a company developing RNA rewriting (or RNA exon editing) technology, announced a research collaboration and licensing agreement with Roche for the discovery and development of therapeutics targeting neurological diseases. Ascidian designs and develops therapeutics that edit RNA exons at the kilobase scale. The deal brings together RNA Exon Editors with Roche’s CNS delivery capabilities to develop novel genomic medicines. Ascidian will receive $42 million in initial payment, and up to $1.8 billion in research, clinical, and commercial milestone payments, as well as commercial royalties.

“Current gene therapy and gene editing approaches to neurological conditions have several challenges,” noted Michael Ehlers, MD, PhD, interim president & CEO, Ascidian Therapeutics. With Ascidian’s technology, he continued, “a single exon editing molecule can be used to replace multiple mutated exons simultaneously without any foreign enzyme through pre-mRNA trans-splicing. The resulting edited mRNA is expressed using the native gene expression circuitry of the cell and can be packaged into a single AAV for in vivo delivery.”

Ascidian’s platform enables targeting of large genes, and genes with high mutational variance, while maintaining native gene expression patterns and levels. RNA exon editing excises mutated or disease-causing exons and replaces them with wild-type exons. A single RNA exon editor can be used to remove and replace multiple whole exons simultaneously, in a single reaction, without any DNA edits and without the use of any foreign enzymes.

Ascidian’s exon editing technology is designed to provide the durability of gene therapy, while reducing risks associated with direct DNA editing and gene replacement. The company noted that the exon editing payload is small enough to fit in AAV or other viral or nonviral delivery vehicles, including lipid nanoparticles, because only disease-causing exons in a gene need to be replaced.

Earlier this year, Ascidian announced FDA IND clearance for the first-ever RNA exon editing candidate, ACDN-01, which targets Stargardt disease and other ABCA4 retinopathies. Ascidian is currently executing the Phase I/II STELLAR clinical trial to evaluate the safety and efficacy of ACDN-01.

Under the agreement, Ascidian will provide Roche exclusive, target-specific rights to Ascidian’s RNA exon editing technology for undisclosed neurological targets. Ascidian will conduct discovery and certain preclinical activities in collaboration with Roche, and Roche will be responsible for certain preclinical activities, and further clinical development, manufacturing, and commercialization. Based on the terms of the agreement, Ascidian is free to develop programs against other neurological targets internally or with other collaborators.

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