{"id":35027,"date":"2025-03-25T11:00:00","date_gmt":"2025-03-25T11:00:00","guid":{"rendered":"https:\/\/www.prnasia.com\/story\/archive\/4649191_CN49191_0"},"modified":"2025-03-25T11:00:00","modified_gmt":"2025-03-25T11:00:00","slug":"accuredit-therapeutics-lnp-based-in-vivo-gene-editing-product-art001-obtained-orphan-drug-designation-from-the-fda","status":"publish","type":"post","link":"https:\/\/transmediavictoria.net.au\/?p=35027","title":{"rendered":"AccurEdit Therapeutics&#8217; LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA"},"content":{"rendered":"<table name=\"logo_release\" border=\"0\" cellspacing=\"10\" cellpadding=\"5\" align=\"right\">\n<tbody>\n<tr>\n<td><img decoding=\"async\" src=\"https:\/\/mma.prnasia.com\/media2\/1725691\/Logo.jpg?p=medium600\" border=\"0\" alt title=\"logo\" hspace=\"0\" vspace=\"0\" width=\"118\"><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p><span class=\"legendSpanClass\"><span class=\"xn-location\">CAMBRIDGE, Mass.<\/span><\/span>, <span class=\"legendSpanClass\"><span class=\"xn-chron\">March 25, 2025<\/span><\/span> \/PRNewswire\/ &#8212; AccurEdit Therapeutics today announced that its innovative <i><span id=\"spanHghlta6c0\">in <\/span><\/i><i><span id=\"spanHghlt5467\">vivo<\/span> <\/i>gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).<\/p>\n<p>ART001, which utilizes lipid nanoparticle (LNP) technology for targeted gene editing, is designed for the treatment of transthyretin amyloidosis (ATTR), a rare and life-threatening disease. It is currently the first and only LNP-based in<i> vivo <\/i>gene editing product worldwide that has cleared an IND in both <span class=\"xn-location\">China<\/span> and <span class=\"xn-location\">the United States<\/span>. It was also <span class=\"xn-location\">China&#8217;s<\/span> first LNP-based in<i> vivo <\/i>gene editing product to enter clinical study in human in <span class=\"xn-chron\">August 2023<\/span>. Clinical data shows that a single dose of ART001 can achieve a &gt;90% reduction of transthyretin (TTR) in the serum. The level of reduction has remained stable for more than 15 months by now, exceeding the natural renewal cycle of the human liver. This is the first, and by far the only, evidence obtained in <span class=\"xn-location\">China<\/span> to confirm the potential of<i> in vivo<\/i> gene editing therapy such as ART001 as a one-time, lifelong treatment.<\/p>\n<p>Other<i> in vivo<\/i> gene editing products developed in the US and <span class=\"xn-location\">China<\/span> have reported infusion-related reactions frequently in their clinical trials, including severe cases that require emergency care. No infusion-related reactions were observed in patients enrolled in the first clinical study of ART001 with at least 15 months follow up. Additionally, no off-target editing was detected even at doses tens of times higher than the saturating dose of ART001 in primary human hepatocytes.<\/p>\n<p>Dr. <span class=\"xn-person\">Yongzhong Wang<\/span>, Founder, Chairman, and CEO of AccurEdit Therapeutics, stated, &#8220;The orphan drug designation for ART001 by the FDA provides a solid foundation for its global development. Our team is excited to have the opportunity to develop ART001 as a safe, effective, and accessible treatment option for ATTR patients around the world.&#8221;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>CAMBRIDGE, Mass., March 25, 2025 \/PRNewswire\/ &#8212; AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). ART001, which utilizes lipid nanoparticle (LNP) technology for targeted gene editing, is designed for the treatment of transthyretin amyloidosis (ATTR), a [&hellip;]<\/p>\n","protected":false},"author":8,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[20],"tags":[],"class_list":["post-35027","post","type-post","status-publish","format-standard","hentry","category-business-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.9 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>AccurEdit Therapeutics&#039; LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA - My Blog<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/transmediavictoria.net.au\/?p=35027\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"AccurEdit Therapeutics&#039; LNP-based In Vivo Gene Editing Product ART001 Obtained Orphan Drug Designation from the FDA - My Blog\" \/>\n<meta property=\"og:description\" content=\"CAMBRIDGE, Mass., March 25, 2025 \/PRNewswire\/ &#8212; AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). 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AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). 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