{"id":6552,"date":"2026-06-20T16:57:48","date_gmt":"2026-06-20T11:57:48","guid":{"rendered":"https:\/\/cifrum.kz\/genetic-breakthrough-first-in-vivo-crispr-therapy-succeeds-in-historic-phase-3-trial\/"},"modified":"2026-06-20T16:57:48","modified_gmt":"2026-06-20T11:57:48","slug":"genetic-breakthrough-first-in-vivo-crispr-therapy-succeeds-in-historic-phase-3-trial","status":"publish","type":"post","link":"https:\/\/cifrum.kz\/en\/genetic-breakthrough-first-in-vivo-crispr-therapy-succeeds-in-historic-phase-3-trial\/","title":{"rendered":"GENETIC BREAKTHROUGH: First In Vivo CRISPR Therapy Succeeds in Historic Phase 3 Trial"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\"><strong>ISTANBUL \/ CAMBRIDGE, MA<\/strong> \u2014 An international team of scientists has announced the completion of the first-ever large-scale Phase 3 clinical trial of a CRISPR gene-editing treatment delivered directly inside the human body. The results, presented at the European Academy of Allergy and Clinical Immunology (EAACI) congress, confirm that a single infusion can potentially cure a rare genetic disease.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Success Statistics<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">The randomized, double-blind study involved 80 patients with hereditary angioedema (HAE) \u2014 a life-threatening condition characterized by unpredictable and severe swelling. The investigational therapy, <strong>lonvoguran-ziclumeran (lonvo-z)<\/strong> by Intellia Therapeutics, demonstrated phenomenal efficacy:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>The rate of monthly attacks was reduced by <strong>87%<\/strong> compared to the placebo group.<\/li>\n<li><strong>62%<\/strong> of treated patients remained entirely attack-free after a single dose.<\/li>\n<li>The need for on-demand rescue therapy dropped by <strong>89%<\/strong>.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">Expert Insights<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">&#8220;This is the first time CRISPR technology has been successfully applied systemically within a large Phase 3 trial. We are seeing more than just improvement; we are seeing the potential for a one-time functional cure for a chronic condition,&#8221; said Dr. Danny Cohn of Amsterdam University Medical Centers (UMC), who led the research.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The findings were simultaneously published in the prestigious <em>The New England Journal of Medicine (NEJM)<\/em>. Researchers emphasized the safety profile of the method: side effects were mostly mild and transient, and long-term follow-up from Phase 1 and 2 trials confirms the effect remains stable for over four years.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">What Does This Mean for Medicine?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Intellia Therapeutics&#8217; success paves the way for the regulatory approval of the world&#8217;s first <em>in vivo<\/em> gene-editing drug. Unlike previous &#8220;ex vivo&#8221; methods, where a patient&#8217;s cells are edited in a lab and then re-infused, this new approach allows DNA to be &#8220;fixed&#8221; directly in the patient&#8217;s liver through a standard intravenous infusion.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Experts believe this marks the beginning of a new era in treating thousands of hereditary disorders that were previously considered incurable.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><em>Source: <a href=\"https:\/\/ir.intelliatx.com\/news-releases\/news-release-details\/intellia-therapeutics-reports-additional-positive-phase-3\" target=\"_blank\" rel=\"noopener\">Intellia Therapeutics<\/a>, <a href=\"https:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2600931\" target=\"_blank\" rel=\"noopener\">NEJM<\/a><\/em><\/p>\n\n","protected":false},"excerpt":{"rendered":"<p>ISTANBUL \/ CAMBRIDGE, MA \u2014 An international team of scientists has announced the completion of the first-ever large-scale Phase 3 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6549,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center 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