KLK14

CAMBRIDGE, Mass., Oct. 31, 2022 (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 that an abstract featuring new interim clinical data from the Phase 1/2 study of NTLA-2002 has been selected for a Distinguished Industry Abstract oral presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting, taking place November 10 – 14 in Louisville, Kentucky. NTLA-2002 is an investigational in vivo CRISPR/Cas9 therapy in development as a single-dose treatment to prevent angioedema attacks in people living with hereditary angioedema (HAE). Building on data released at the 2022 Bradykinin Symposium, the presentation will include additional interim safety, kallikrein reduction and attack rate data from the ongoing dose-escalation portion of Intellia’s first-in-human study of NTLA-2002. Presentation Details Title: "In vivo CRISPR/Cas9 editing of KLKB1 in Patients with Hereditary Angioedema: A First-in-Human Study” Date and Time: Saturday, November 12, 2022, from 4:30 – 5:30 p.m. ET Session: Distinguished Industry Oral Abstracts - Session A Presenter: Dr. Hilary Longhurst, M.D., Ph.D., FRCP, FRCPath, Senior Medical Officer at Auckland District Health Board and Honorary Associate Professor at University of Auckland, New Zealand, the trial’s principal investigator in New Zealand Intellia Therapeutics Investor Event Intellia will host a live webcast on Monday, November 14, 2022, at 8:00 a.m. ET to review the presented data. To join the webcast, please visit this link, or the Events and Presentations page of the Investors & Media section of the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least 30 days following the call. About the NTLA-2002 Clinical Program Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies. Visit clinicaltrials.gov (NCT05120830) for more details. About NTLA-2002 Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. 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 together carry out the precision editing. About Hereditary Angioedema Hereditary angioedema (HAE) is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks. 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 intelliatx.com. Follow us on Twitter @intelliatx.

CAMBRIDGE, Mass., Sept. 08, 2022 (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 that it will host a virtual investor event to present interim data from the first-in-human clinical studies of NTLA-2002 and NTLA-2001 on September 16, 2022, at 8:00 a.m. ET. NTLA-2002 First Interim Clinical Data Review Intellia will review the interim clinical data from the Phase 1/2 study of NTLA-2002 for the treatment of hereditary angioedema (HAE), which is scheduled to be presented at the 2022 Bradykinin Symposium on September 16 in Berlin, Germany. The presentation will focus on the initial safety, kallikrein protein reduction and HAE attack rate data from the dose-escalation portion of the study. NTLA-2002 is Intellia’s wholly owned, second systemically administered in vivo CRISPR candidate. NTLA-2001 Interim Clinical Data Update from the Cardiomyopathy Arm Intellia will present interim clinical data from the ongoing Phase 1 study of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. The study update will focus on the dose-escalation portion of the cardiomyopathy arm of the study. The event will include a presentation by 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 national coordinating investigator. NTLA-2001, which is being developed as part of a multi-target collaboration with Regeneron, is being evaluated in patients with either ATTR amyloidosis with polyneuropathy (ATTRv-PN) or ATTR amyloidosis with cardiomyopathy (ATTR-CM). To join the webcast on September 16, 2022, at 8:00 a.m. ET, please visit this link, or the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com. A replay of the events will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website for at least 30 days following the event. About NTLA-2002 Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. 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 together carry out the precision editing. 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 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. 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 intelliatx.com. Follow us on Twitter @intelliatx.