Lysogene宣布首位患者使用lysg - gm101实验性基因疗法治疗GM1神经节脂肪沉积症亚博登录首页亚搏全站app下载

Lysogene宣布首位患者使用lysg - gm101实验性基因疗法治疗GM1神经节脂肪沉积症亚博登录首页亚搏全站app下载150.150.Lysogene
  • 二溶基因CNS基因治疗方案进入临床亚搏全站app下载
  • 正在对英国、美国和法国总共16名患者进行研究

法国巴黎- 2021年6月9日下午06:00 - Lysogene (FR0013233475 - LYS),英国皇家曼彻斯特儿童亚搏全站app下载医院(Royal Manchester Children’s Hospital,隶属于曼彻斯特大学NHS Foundation Trust)的一家针对中枢神经系统(CNS)疾病的3期基因治疗平台公司今天宣布,在全球适应设计临床试验中,为首名患者使用LYS-GM101 (NCT04273269)。一种亚搏全站app下载治疗GM1神经节脂肪增多症的基因疗法。亚博登录首页

本试验是一项介入性、多中心、单臂、两阶段适应性设计研究,评估重组腺相关病毒载体rh - 10血清型的胸腔内传递(AAVrh.10)携带人β-半乳糖苷酶基因(GBL1)。临床试验包括一个安全性阶段和一个验证有效性阶段。该试验将在美国和欧洲招募16名诊断为早期或晚期婴儿GM1神经节苷脂病的患者。亚博登录首页更多信息可在www.clinicaltrials.gov。

亚博登录首页GM1 GALISICOSIS是由GLB1基因突变引起的致命常染色体隐性疾病,导致在神经元中的GM1神经节苷脂积累导致逐步神经变性。到目前为止,迄今为止没有治疗批准。

“在这项临床研究中,给第一个患者注射药物代表了Lysogene的一个重要里程碑,因为它标志着我们的第二个基因治疗项目进入了临床。亚搏全站app下载这再次表明,尽管我们最近面临Covid-19的挑战,但我们有执行能力“ 说过Karen Aiach,创始人董事长兼赖戈纳州首席执行官“我们一直在寻找加速新型基因治疗治疗的发展,以改善患者的生命,并兴奋地将该调查疗法带给GM1神经病症的患者。亚博登录首页亚搏全站app下载

G亚博登录首页M1神经节osidosis是一种破坏性的、进展迅速的疾病,尤其是在最严重的情况下,它会导致严重的神经退化和过早死亡。, 说过西蒙·琼斯,医学博士,皇家曼彻斯特儿童医院儿科遗传代谢病首席研究员和顾问,曼彻斯特大学NHS基金会信托(MFT)的一部分,曼彻斯特大学高级讲师。“我很高兴开始用Lys-Gm101注射第一个患者,其行政当局完全良好”。

LYS-GM101('腺相关病毒载体rh血清型。10 expressing beta-galactosidase’) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as the Rare Pediatric Disease designation in the US in 2016.

Lysogene还资助GM1甘道菌病的自然历史研究由C亚博登录首页asimir试验进行Casimir试验,收集做出某些日常任务和行为的儿童的预期和/或回顾视频(NCT04310163).

关于Lysogene

Lysogene是一家专注于亚搏全站app下载中枢神经系统孤儿病治疗的基因治疗公司。该公司建立了一种独特的能力,能够安全有效地将基因疗法传递到中枢神经系统,以治疗溶酶体疾病和中枢神经系统的其他遗传疾病。与Sarepta Therapeutics, Inc.合作的MPS IIIA 2/3期临床试验正在进行中,GM1神经节osidosis的1/3期临床试验正在准备中。亚博登录首页根据Lysogene与Sarepta Therapeutics, Inc.签署的协议,Sarepta Therapeutics, Inc.将持有LYS-SAF302在美国和欧洲以外市场的独家商业权利;和Lysogene将保持LYS-SAF302在欧洲的商业专有权。Lysogene还与一个学术伙伴合作,确定治疗脆性X综合征(一种与自闭症相关的遗传疾病)的发展战略。www.astridbowlby.com

关于曼彻斯特大学NHS基金会信任

曼彻斯特大学NHS信托基金是英国最大的NHS信托基金之一,是专业医疗保健服务的领先提供商。它的10家医院拥有数百名世界一流的临床医生和学术人员,致力于为患者提供最好的护理和治疗。
它的医院有曼彻斯特皇家医院、圣玛丽医院、曼彻斯特儿童医院、曼彻斯特皇家眼科医院、曼彻斯特大学牙科医院、特拉福德综合医院、阿尔特林查姆医院、威森肖医院、威辛顿医院和北曼彻斯特综合医院。
更多信息可用www.mft.nhs.uk

前瞻性陈述

本新闻稿可能包含某些前瞻性陈述,特别是本公司对其临床试验和现金跑道的进展。Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Company’s control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Company’s regulatory filings with the French Autorité des Marchés Financiers, including in the 2020 universal registration document, registered with the French Markets Authorities on April 12, 2021, under number D.21-0296, and future filings and reports by the Company. Furthermore, theseforward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

本新闻稿以法语和英语编写。如果两者之间有任何差异
案文中,法语本应取代。

联系人
Stéphanedurant des aulnois
首席财务官
stephane.durant-des-aulnois@lysogene.com
+33 1 41 43 03 99

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