Intellia Therapeutics Receives Orphan Drug Designation from US FDA for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin Amyloidosis (ATTR)


CAMBRIDGE, Mass., October 21, 2021 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage genome editing company focused on developing curative treatments using CRISPR / Cas9 technology at the times in vivo and ex vivo, today announced that the United States Food and Drug Administration (FDA) has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR). This experimental therapy is the first CRISPR therapy to be administered systemically to modify a pathogenic gene inside the human body. NTLA-2001 has the potential to be the first single dose treatment for ATTR amyloidosis because it may be able to stop and reverse the devastating complications of this disease. ATTR amyloidosis is a rare disease that can affect a number of organs and tissues in the body by the build-up of misfolded transthyretin protein (TTR) deposits.

“Orphan drug designation underscores FDA recognition of the potential promise of NTLA-2001 as a novel single-dose therapy for the treatment of ATTR amyloidosis,” said Intellia President and CEO , John Leonard, MD. “At Intellia, we are committed to advancing our modular genome editing platform to develop potentially curative treatment options for life-threatening diseases, and we look forward to working with the ATTR amyloidosis community and the FDA to provide much needed treatment option for patients.

NTLA-2001 is currently being investigated in a phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). In June 2021, Intellia and its collaborator Regeneron announcement positive intermediate clinical results of the first two cohorts of this study. These results, which were published in the New England Journal of Medicine, represented the earliest clinical data supporting the safety and efficacy of in vivo Editing of the CRISPR genome in humans.

The FDA’s Orphan Drug Designation Program confers orphan status on drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. The orphan drug designation qualifies the drug sponsor for certain development incentives, including tax credits for qualified clinical trials, prescription drug user fee exemptions, and marketing exclusivity for seven years after approval. of the FDA. The FDA decision follows a March 2021 decision by the European Commission (EC) to also grant orphan drug designation NTLA-2001 for the treatment of ATTR amyloidosis.

About transthyretin amyloidosis (ATTR)
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR amyloidosis (ATTRv) occurs when a person is born with mutations in the TTR gene, which causes the liver to produce a structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid deposits in the body, causing serious complications in several tissues, including the heart, nerves, and digestive system. ATTRv amyloidosis mainly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without any genetic mutations produce unmutated or wild-type TTR proteins which become unstable over time, folding poorly and aggregating into disease-causing amyloid deposits. This condition, called wild-type ATTR amyloidosis (ATTRwt), primarily affects the heart.

About NTLA-2001
Based on Nobel Prize-winning CRISPR / Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR amyloidosis. NTLA-2001 is the first experimental candidate for CRISPR therapy to be administered systemically or intravenously to alter genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to provide the liver with a two-part genome-editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the disease. enzyme Cas9, which performs precision editing. Strong preclinical data, showing deep and lasting reduction in transthyretin (TTR) after in vivo inactivation of the target gene supports the potential of NTLA-2001 as a single dose therapy. Provisional phase 1 clinical data published in June 2021 confirms a substantial and dose-dependent reduction in TTR protein after a single dose of NTLA-2001. Intellia leads the development and commercialization of NTLA-2001 through multi-target research, development and commercialization collaboration with Regeneron.

About Intellia Therapeutics
Intellia Therapeutics, one of the leading clinical-stage genome editing companies, is developing new potentially curative therapies using CRISPR / Cas9 technology. To fully realize the transformational potential of CRISPR / Cas9, Intellia pursues two main approaches. Of the society in vivo The programs use intravenously administered CRISPR as therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly in 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 experience in scientific, technical and clinical development, as well as its strong intellectual property portfolio, has enabled the company to play a leadership role in harnessing the full potential of CRISPR / Cas9 to create new classes of genetic medicine. Learn more about Follow us on twitter @intelliatweets.

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. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding its: being able to complete clinical studies for NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”) in accordance with its trial requests clinical (“CTA”), including the submission of regulatory applications in other countries; ability to demonstrate the efficacy of NTLA-2001 in the treatment or reversal of ATTR amyloidosis in patients; advancing and expanding its CRISPR / Cas9 technology to develop human therapeutics, as well as its ability to maintain and expand its related intellectual property portfolio; expectations regarding the potential impact of the 2019 coronavirus disease pandemic on the strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including, but not limited to, its collaborations with Regeneron, including its co-development programs for ATTR amyloidosis; and statements regarding the timing of regulatory filings regarding its development programs.

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 unfavorably to those stated or implied by such forward-looking statements. forward-looking 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 associated with 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 associated with the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that one or more of Intellia’s product candidates will not be successfully developed and marketed; the risk that the results of preclinical studies or clinical studies are not predictive of future results in relation to future studies; and the risk that Intellia’s collaborations with Regeneron or its other collaborations will not continue or be successful. For a discussion of these risks and uncertainties, as well as other important factors, any of which could cause Intellia’s actual results to differ from those contained in 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 other documents filed by Intellia with the Securities and Exchange Commission (“SEC”). All information in this press release is as of the date of publication, and Intellia does not undertake to update this information, except as required by law.

Intellia contacts:

Ian karp
Senior Vice-President, Investor Relations and Corporate Communications
+ 1-857-449-4175
[email protected]

Lina li
Director, Investor Relations
+ 1-857-706-1612
[email protected]

Lisa Qu
Ten communication bridges
+ 1-678-662-9166
[email protected]
[email protected]

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