Intellia Therapeutics to Present New Preclinical Data from Its CRISPR/Cas9 Programs at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy
Intellia’s data include important updates about the company’s progress and platform development activities:
“Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering”
As a follow-on to data presented at Keystone Symposia’s Engineering the
Abstract number: 35
Session: CAR T-Cell Therapies I
“Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia”
This presentation will focus on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to apply CRISPR/Cas9 technology to develop engineered T cell therapies to address intractable cancers, such as AML. Researchers will also present data showing a significant decrease in AML tumor burden in mice treated with the WT1-specific TCRs, which are incorporated into Intellia’s lead NTLA-5001 candidate for the treatment of AML.
Abstract number: 78
Session: CAR T and Other Engineered T Cells Targeting Hematological Malignancies
“CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema”
Intellia will present data updates on its potential hereditary angioedema (HAE) therapy, which uses the company’s modular lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system to knock out the prekallikrein B1 (KLKB1) gene and reduce kallikrein activity. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. This presentation will include results from the company’s ongoing collaboration with researchers at Regeneron and also will build on initial data released at Keystone Symposia’s Engineering the
Abstract number: 1320
Session: Gene Regulation and Delivery Technologies
“Validation of CRISPR/Cas9 Off-Target Discovery Profiles from In Silico Prediction, Cell-Based and Biochemical-Based Assays with Targeted Off-Target Sequencing”
This presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.
Abstract number: 203
Researchers will present improved murine models used to assess safety, efficacy and persistence of therapeutic modalities in cell-based therapies. Together with biotechnology company Taconic Biosciences, Intellia developed two in vivo mouse models, which allow for enhanced monitoring of graft-versus-host disease and human natural killer cytotoxicity.
Abstract number: 1174
Workshop and Symposia
“Building a Modular CRISPR/Cas9 Platform for Human Therapeutic Applications”
Intellia will participate in the
Industry Sponsored Symposia
Intellia will participate in an industry lunchtime panel discussion during ASGCT.
Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients’ diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.
This press release contains “forward-looking statements” of
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Regeneron, OSR or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the
External Affairs & Communications
Source: Intellia Therapeutics, Inc.