Intellia Therapeutics in Prese –

  • Oral presentation to include additional data on patient durability treated with NTLA-2001, the first-ever systemically administered live Experimental CRISPR therapy
  • Submission must include data supporting fixed-dose selection into the ongoing single-dose expansion cohort of the polyneuropathy arm

CAMBRIDGE, Mass., June 08, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NTLA, Financial), a leading genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that interim clinical data from the Phase 1 study of NTLA-2001 , an investigational therapy in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, will be shared in an oral presentation at the European Association for the Study of the Liver™ 2022 International Liver Congress, taking place from June 22-26 in London.

“We are pleased to present longer-term follow-up data from the first-in-man study of NTLA-2001, which is designed to potentially halt progression and reverse ATTR amyloidosis,” said John Leonard, President- CEO of Intellia. , MD “We look forward to sharing interim data that we believe demonstrates the potential of our experimental CRISPR-based therapy as a single-dose treatment that provides a profound and long-lasting response.”

The presentation will include new data from the ongoing Phase 1 study evaluating NTLA-2001 in people with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). These include additional results from the four dose-escalation cohorts in Part 1, highlighting the durability of response after a single dose of NTLA-2001. Data supporting fixed dose selection for Part 2, the single-dose expansion cohort of the polyneuropathy arm, will also be presented.

The presentation will build on interim data presented in February, which showed significant reductions in serum TTR levels from baseline in a dose-dependent manner in 15 patients with vATTR-PN. At the highest dose tested, 1.0 mg/kg, the mean and maximum reductions in serum TTR were 93% and 98%, respectively, on day 28 in the six treated patients. The company also previously announced that it is now evaluating a fixed dose of 80 mg in Part 2 of the Phase 1 study, which is expected to provide similar exposure to the 1.0 mg/kg dose in the vATTR target population. -PN, and that the first patient was dosed in the expansion cohort in the polyneuropathy arm. Intellia is also continuing to dose patients in the cardiomyopathy arm of the Phase 1 study, which is evaluating NTLA-2001 in dose-escalation cohorts of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM).

Presentation details

Title: invivo CRISPR/Cas9 edition of TTR gene with NTLA-2001 in patients with transthyretin amyloidosis – dose selection considerations

Session: Rare liver diseases (including pediatric and genetic)

Date and time: Friday June 24, 2022 from 10:00 a.m. to 11:30 a.m. BST

Presenter: Dr Edward J. Gane, MBChB, MD, FRACP, MNZM, Professor of Medicine at the University of Auckland, New Zealand and Chief Hepatologist, Transplant Physician and Deputy Director of the New Zealand Liver Transplant Unit at the Auckland Hospital, New Zealand’s National Investigator Trial

Intellia Therapeutics Investor Event and Webcast Information

Intellia will host a live webcast on Friday, June 24, 2022 at 8:00 a.m. ET to review the data presented. To participate in the webcast, please visit this link or the Events & Presentations page in the Investors & Media section of the Company’s website at A replay of the webcast will be available on Intellia’s website for at least 30 days after the call.

About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR amyloidosis (vATTR) occurs when a person is born with mutations in the TTR , which causes the liver to produce a structurally abnormal protein transthyretin (TTR) with a propensity to misfold. These damaged proteins build up as amyloid in the body, leading to serious complications in many tissues, including the heart, nerves and digestive system. VATTR amyloidosis primarily manifests as polyneuropathy (vATTR-PN), which can lead to nerve damage, or cardiomyopathy (vATTR-CM), which can lead to heart failure. Some people without genetic mutation produce unmutated or wild-type TTR proteins that become unstable over time, fold, and aggregate into pathogenic amyloid deposits. This condition, called wild-type ATTR amyloidosis (ATTRwt), primarily affects the heart. There are an estimated 50,000 people worldwide with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis.

About NTLA-2001
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first experimental CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver a two-part genome-editing system to the liver: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which performs precision editing. Strong preclinical data, showing profound and long-lasting transthyretin (TTR) reduction after live inactivation of the target gene, supports the potential of NTLA-2001 as a single-dose therapeutic. Intellia is leading the development and commercialization of NTLA-2001 in a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multicenter, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM ). Visit (NCT04601051) for details.

About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing company, is developing potentially curative new therapies by leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two main approaches. From the company live The programs use intravenously administered CRISPR as therapy, in which proprietary delivery technology enables highly precise editing of pathogenic genes directly into specific target tissues. from Intellia ex-vivo the programs use CRISPR to create the therapy using human cells engineered to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical experience, along with its strong intellectual property portfolio, has enabled the company to play a leadership role in harnessing the full potential of genome editing for create new classes of genetic medicine. Learn more about Follow us on Twitter @intelliatx.

Forward-looking statements
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. Such forward-looking statements include, but are not limited to express or implied statements regarding Intellia’s beliefs and expectations regarding: its ability to successfully expand its leadership position and harness the full potential of genomic drugs to enhance its genome editing capabilities and pipeline; the safety, efficacy, success and progress of its clinical programs for NTLA-2001 for the treatment of transthyretin amyloidosis in accordance with its Clinical Trial Applications (“CTAs”), including the anticipated timeline for the publication of data, regulatory filings and the initiation, registration, and completion of clinical trials; expansion of its CRISPR/Cas9 technology and related technologies to advance additional development candidates and timing expectations for advancing such development candidates; its ability to maintain and develop its related intellectual property portfolio; expectations regarding the potential impact of the coronavirus disease pandemic, including the impact of any variants, on the strategy, future operations and timing of its clinical trials; its ability to optimize the impact of its collaborations on its development programs, including, but not limited to, its collaboration with Regeneron Pharmaceuticals, Inc.

All forward-looking statements contained in this press release are based on management’s current expectations and beliefs regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially. and adversely from those stated or implied by such statements. – look at the statements. These risks and uncertainties include, but are not limited to: risks relating to Intellia’s ability to protect and maintain its intellectual property position; risks relating 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 relating to the authorization, initiation and conduct of studies and other development requirements for its product candidates, including uncertainties relating to regulatory approvals to conduct clinical trials; the risk that 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 future studies; the risk that the results of clinical studies will not be positive; and the risk that Intellia’s collaborations with Regeneron or its other collaborations may not continue or be successful. For a discussion of these and other risks and uncertainties, and other important factors, each 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 contained in this press release is as of the date of publication, and Intellia undertakes no obligation to update this information, except as required by law.

Intellia contacts:

Ian Karp
Senior Vice President, Investor Relations and Corporate Communications
[email protected]

Lina Li
Director, Investor Relations and Corporate Communications
[email protected]

Matt Crenson
Ten-bridge communications
[email protected]
[email protected]



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