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Shape Therapeutics RNAfix® Editing Platform Achieves >90% Editing in Mouse and Non-Human Primate Brain

May 08, 2024 at 09:31 AM EDT

ⓘ This article is third-party content and does not represent the views of this site. We make no guarantees regarding its accuracy or completeness.

RNAfix achieves up to 92% editing in the non-human primate brain after AAV-based gRNA delivery

RNAfix achieves >95% editing throughout the mouse brain with sustained durability at least 6 months post-dose

RNAfix provides a best-in-class safety profile in genomic medicine

SEATTLE, May 08, 2024 (GLOBE NEWSWIRE) -- Shape Therapeutics Inc. (ShapeTX), a leader at the convergence of AI and RNA editing to revolutionize genomic medicine, today presented data at the 27th American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, MD showcasing the high therapeutic potential of its RNAfix platform in for the treatment of central nervous system (CNS) disorders.

The Company will be releasing the data in an oral presentation titled “>95% targeted RNA editing in total brain with high durability and specificity enabled by systemic injection of AAV-packaged ADAR guide RNAs.” The presentation will highlight best-in-class RNA editing efficiency in vivo in mouse, non-human primate (NHP) and in human neuronal model systems. Potential therapeutic applications of RNAfix will be highlighted in Rett syndrome, Parkinson’s disease, and Alzheimer’s disease, showcasing a diversity of therapeutic mechanisms enabled by nucleotide-level precision RNA editing. A superior safety profile will also be presented, with no significant off-target editing across the entire transcriptome. These findings demonstrate a breakthrough in the potential for translating RNAfix into human clinical studies and represent significant advancements in the field of genomic medicine.

David Huss, PhD, Chief Scientific Officer at ShapeTX commented, “RNAfix is efficient, specific, durable, and tunable. The data presented at ASGCT highlight levels of editing in the CNS unparalleled by any other editing technology, unlocking vast therapeutic opportunities. Importantly, the RNAfix platform leaves genomic DNA intact and does not require the delivery of foreign proteins with immunogenicity risks. These significant scientific advancements provide a clear path forward and give us confidence as we build transformative therapies for patients in need.”

About ShapeTX
Shape Therapeutics operates at the convergence of AI and RNA editing to revolutionize genomic medicine. Our proprietary RNA editing technology, RNAfix®, represents a significant leap forward over CRISPR-based tools, achieving 95% editing efficiency in vivo, and a path to one-time curative treatments for thousands of diseases. With over 90 million laboratory-validated guide RNA and 12 billion NHP-derived AAV data points, ShapeTX is building the largest therapeutic RNA foundation model in the industry. Having secured strategic collaborations worth over $4 billion with industry-leading pharmaceutical firms, ShapeTX is poised for substantial growth and to cement our position as a leader in AI for genomic medicine. You can find us at ShapeTX.com and on LinkedIn and X.

Contact:
Lisa Taylor Ash
lisa@shapetx.com