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Intellia Therapeutics to Present Updated Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis and Hold Conference Call to Discuss Third Quarter 2023 Earnings in November

October 26, 2023
  • New interim NTLA-2001 clinical data to be presented at the 4th International ATTR Amyloidosis Meeting on November 2; data to include safety and both absolute and percent change in serum TTR reduction from cardiomyopathy and polyneuropathy arms of the study
  • Intellia management to discuss latest NTLA-2001 data and share additional information about the upcoming Phase 3 study design at Q3 earnings webcast on November 9 at 8 a.m. ET     

CAMBRIDGE, Mass., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced two upcoming events in November.

NTLA-2001 Interim Clinical Data Update
Intellia will present updated data from the ongoing NTLA-2001 Phase 1 study for the treatment of transthyretin (ATTR) amyloidosis being presented at the 4th International ATTR Amyloidosis Meeting, taking place November 2 – 3 in Madrid, Spain.

  • Presentation Details:
    • Title: "Enabling the Development of Serum TTR as a Biomarker for Treatment of ATTR Amyloidosis
    • Date and Time: Thursday, November 2, 2023, from 11:30 a.m.1:00 p.m. CET
    • Session: Latest Data for Patients’ Diagnosis in a Changing World
    • Presenter: Dr. Julian Gillmore, M.D, Ph.D., FRCP, FRCPath, Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K., the trial’s U.K. national coordinating investigator

Third Quarter 2023 Earnings
Intellia will host a webcast to discuss its third quarter 2023 financial results and operational highlights on November 9, 2023, at 8 a.m. ET. The Company will also review the NTLA-2001 data presented at the 4th International ATTR Amyloidosis Meeting.

  • To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section on Intellia’s website at
  • A replay of the call will be available beginning on November 9, 2023, at 12 p.m. ET.

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 investigational 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 to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical and clinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The trial is now closed for enrollment. Visit (NCT04601051) for more details.

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

About Intellia Therapeutics

Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine. Learn more at Follow us on X (formerly known as Twitter) @intelliatx.

Intellia Contacts:

Ian Karp
Senior Vice President, Investor Relations and Corporate Communications

Lina Li
Senior Director, Investor Relations and Corporate Communications

Matt Crenson
Ten Bridge Communications

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Source: Intellia Therapeutics, Inc.