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Intellia Therapeutics Presents New Preclinical Data Showing Persistent In Vivo Editing and Durability of Effect Following CRISPR/Cas9-Based Treatment
In Vivo gene knockout and insertion data to be presented at OTS Annual Meeting highlight modularity of Intellia’s platform and potential for variety of
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"In Vivo gene knockout and insertion data to be presented at OTS Annual Meeting highlight modularity of Intellia’s platform and potential for variety of single-course therapies, with company’s first systemic treatment (NTLA-2001) expected to enter the clinic by year-end\n Liver insertion platform shows promise as best-in-class targeted gene insertion approach to durably restore functional protein, compared to traditional gene therapy CAMBRIDGE, Mass., Sept. 29, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA) is presenting new data demonstrating the persistence of in vivo CRISPR/Cas9 edits to either reduce a disease-causing protein or restore a functional protein, in a mouse model of accelerated liver regeneration. These data will be included in the company’s invited talk at this year’s 16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), taking place virtually from September 27-30, 2020. “As we prepare to enter the clinic with NTLA-2001, our first systemic treatment, we are extremely encouraged by the durability Intellia scientists observed with both gene knockout as well as targeted insertion in a partial hepatectomy (PHx) mouse model. The persistence of these edits and durable effects further support our technology’s ability to develop potentially curative single-course therapies, and provide clear differentiation from chronic treatments and traditional AAV gene therapy,” said Intellia Chief Scientific Officer Laura Sepp-Lorenzino, Ph.D. “Our modular delivery platform is enabling us to rapidly advance multiple product candidates in parallel – and to ensure that the therapeutic impact will be long-lasting for patients in need.” The OTS talk titled, “A Modular CRISPR/Cas9 Genome Editing Platform for Durable Therapeutic Knockout and Targeted Gene Insertion Applications,” will be given today at 10 a.m. ET by Anthony Forget, Ph.D., senior director of genome editing at Intellia. Click here to view the presentation slides. Persistent In Vivo Liver Gene Knockouts and Corresponding Protein Reduction Achieved Employing Intellia’s Modular Platform Accelerated hepatocyte turnover following PHx in mice was employed to assess the durability of gene knockout and insertion edits. After resection of 2/3 of the liver, and subsequent full-liver regeneration, genome edits and corresponding protein levels were u...