{"id":17412,"date":"2020-06-19T18:03:00","date_gmt":"2020-06-19T16:03:00","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=17412"},"modified":"2020-06-23T18:08:03","modified_gmt":"2020-06-23T16:08:03","slug":"uniqure-announces-first-two-patients-treated-in-phase-i-ii-clinical-trial-of-amt-130-for-the-treatment-of-huntingtons-disease","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/uniqure-announces-first-two-patients-treated-in-phase-i-ii-clinical-trial-of-amt-130-for-the-treatment-of-huntingtons-disease\/","title":{"rendered":"uniQure Announces First Two Patients Treated in Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the first two patients in the Phase I\/II clinical trial of\u00a0AMT-130 for the treatment of Huntington\u2019s disease\u00a0have been treated. The Phase I\/II study is a double-blind, randomized clinical trial being conducted in the United States, with now one patient treated with AMT-130, and one patient who received the imitation surgery.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cFor years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for Huntington\u2019s disease into clinical testing, and this moment marks an important milestone for our company now that we have two AAV gene therapy candidates in clinical development,\u201d said&nbsp;<a href=\"https:\/\/www.globenewswire.com\/Tracker?data=npOsFrG7aX9h5pj41sJYuAUxQqpBo74QTPPy1c2nZ9CJFs5Sdh-ayrMvywpzHWD_1K152phhlZr8G9so-d-RDo8tKB_8ScJ7bTqBHLkfgl31HQbtN4-CHTfDyDB5lcrO\" rel=\"noreferrer noopener\" target=\"_blank\">Matt Kapusta<\/a>, chief executive officer of uniQure. \u201cWith the first two patients treated in this trial, we have taken a significant step forward in advancing AMT-130 closer to our goal of developing a therapy that inhibits the production of the mutant huntingtin protein. We are delighted to be working with leading experts in the field to evaluate this promising candidate.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The\u00a0Phase I\/II clinical trial of AMT-130\u00a0for the treatment of Huntington\u2019s disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntington\u2019s disease randomized to treatment with AMT-130 or an imitation (sham) surgery. The five-year, multi-center trial consists of a blinded 18-month core study period followed by unblinded long-term follow-up. Patients will receive a single administration of AMT-130 through\u00a0MRI-guided, convection-enhanced stereotactic neurosurgical delivery\u00a0directly into the striatum (caudate and putamen). Additional details are available on\u00a0<a rel=\"noreferrer noopener\" href=\"https:\/\/www.globenewswire.com\/Tracker?data=dv7HNHu7Xi2J_oDnm6g-1ps9Hf9BG8Mzk2rPGB6Vqzs0cqrChXbvOk9S__9PfoztQB3yuZT4AOD3ePj-LIlBGbzHgjOlQxXtzfjXvxXLkjE=\" target=\"_blank\">www.clinicaltrails.gov<\/a>\u00a0(NCT04120493).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">AMT-130 is uniQure\u2019s first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE\u2122 platform.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cThere is an urgent need for disease-modifying options to treat Huntington\u2019s disease, and we\u2019re excited to have an investigational gene therapy now available for HD patients,\u201d stated George Yohrling, chief scientific officer and chief mission officer at Huntington&#8217;s Disease Society of America. \u201cBased on the promising preclinical data presented on AMT-130 over the years, we are optimistic about its potential to alter the course of this devastating disease.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>About Huntington\u2019s Disease<\/strong><br>Huntington\u2019s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington\u2019s disease, there are no currently approved therapies to delay the onset or to slow the disease\u2019s progression.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Source: <a href=\"https:\/\/tools.eurolandir.com\/tools\/Pressreleases\/GetPressRelease\/?ID=3781729&amp;lang=en-GB&amp;companycode=nl-qure&amp;v=\"><strong>uniQure<\/strong><\/a> (press release)<\/p>\n","protected":false},"excerpt":{"rendered":"<p>uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the first two patients in the Phase I\/II clinical trial of\u00a0AMT-130 for the treatment of Huntington\u2019s disease\u00a0have been treated. The Phase I\/II study is a double-blind, randomized clinical trial being conducted in the United States, with now [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-17412","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>uniQure Announces First Two Patients Treated in Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease - hollandbio<\/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:\/\/www.hollandbio.nl\/en\/nieuws\/uniqure-announces-first-two-patients-treated-in-phase-i-ii-clinical-trial-of-amt-130-for-the-treatment-of-huntingtons-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"uniQure Announces First Two Patients Treated in Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease - hollandbio\" \/>\n<meta property=\"og:description\" content=\"uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the first two patients in the Phase I\/II clinical trial of\u00a0AMT-130 for the treatment of Huntington\u2019s disease\u00a0have been treated. 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