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Updated preclinical data will be presented on CRISPR/Cas9-mediated targeted gene insertion to treat alpha-1 antitrypsin deficiency (AATD)

CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), today announced the presentation of new data from its CRISPR/Cas9 platform at the 24^th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place virtually from May 11-14, 2021.

“The data accepted for presentation at ASGCT reflects our ongoing commitment to finding new ways to treat and potentially cure a variety of diseases. Utilizing our modular in vivo insertion technology, we demonstrate the potential to durably restore normal AAT protein levels after a single dose. We continue to advance multiple genome editing strategies for patients living with AATD,” said President and Chief Executive Officer John Leonard, M.D. “In a separate presentation, Intellia will share our integrative genomics approach that combines computational, biochemical and cell-based genome-wide off-target discovery as well as characterization of potential DNA structural variants to identify precise CRISPR/Cas9 sequences that are safe human therapeutic candidates.”

ASGCT Annual Meeting Presentations & Invited Sessions

Oral Presentation:

Title: “CRISPR/Cas9-Mediated Targeted Gene Insertion Platform Achieves Durable, Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates”
Abstract number: 15
Session Title: Delivery Technologies and CRISPR for Therapeutics
Session Room: Room 3
Date and Time: Tuesday, May 11, 2021, 5:30 – 5:45 p.m. ET
Presenting Author: Sean Burns M.D., vice president of Intellia’s Disease Biology and Pharmacology group

Invited Talks:

Title: “Characterization of Potential Unintended Genome Editing with CRISPR/Cas9 for New Therapeutics”
Session Type: Pre-Meeting Program
Session Title: Moving Genome Editing to the Clinic: from Technology to Therapeutics
Date and Time: Monday, May 10, 2021, 1:05 – 1:30 p.m. ET
Presenting Author: Daniel O’Connell Ph.D., director of Intellia’s platform development group

Title: “Development of Systemic CRISPR-Based Therapeutics”
Session Title: Genome Editing – Clinical and Preclinical Updates
Date and Time: Tuesday, May 11, 2021, 10:26 – 10:52 a.m. ET
Presenting Author: Laura Sepp-Lorenzino, Ph.D., chief scientific officer

Additional data collected will be included in final meeting presentations. All abstracts for the ASGCT Annual Meeting are available on ASGCT’s website here.

About Intellia Therapeutics
Intellia Therapeutics is a leading clinical-stage genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single administration, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more about Intellia and CRISPR/Cas9 at intelliatx.com. Follow us on Twitter @intelliatweets.

Forward-Looking Statement

This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding its: plans to advance and complete preclinical studies, including non-human primate studies, for its AATD research program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion, as well as knockout editing capabilities; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies, including those its AATD research program, in any future studies, including human clinical trials; and its ability to develop other in vivo or ex vivo cell therapeutics of all types using CRISPR/Cas9 technology. 

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 authorization, 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; and the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies. 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 Securities and Exchange Commission (“SEC”). All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:
Lina Li
Director
Investor Relations
+1-857-706-1612
lina.li@intelliatx.com

Media:
Julie Ferguson
Interim Head of External Affairs & Communications
+1-312-385-0098
julie.ferguson@intelliatx.com

Source: Intellia Therapeutics, Inc.