Lysogene宣布FDA Fast Track指定LYS-GM101基因疗法治疗GM1神经节脂肪病亚博登录首页亚搏全站app下载

Lysogene宣布FDA Fast Track指定LYS-GM101基因疗法治疗GM1神经节脂肪病亚博登录首页亚搏全站app下载150.150.Lysogene

巴黎,法国- 2021年7月8日下午06:00 - Lysogene (FR0013233475 - LYS),基因亚搏全站app下载治疗平台公司针对中枢神经系统(CNS)疾病,今天宣布,美国食品和药物管理局(FDA)给予其LYS-GM101快速跟踪指定计划,目前正在研究在最近发起的全球adaptative-design临床试验治疗GM1 gangliosidosis。亚博登录首页亚博登录首页GM1神经节苷脂病是一种由GLB1基因突变引起的致死性常染色体隐性疾病,导致GM1神经节苷脂在神经元中积聚,导致进行性神经退行性变。迄今为止,还没有批准对这种疾病的治疗方法。

Karen Aiach,创始人,Lysogene董事长和首席执行官评论:“我们很高兴FDA为Lys-Gm101授予了快速的轨道指定,因为它强调其潜力改善GM1神经病症的儿童中的神经认知缺陷,目前没有治疗的致命神经疾病。亚博登录首页我们上个月提出了第一个患者,招聘是在赛道上进行,完成第一季度第一个队列的第一个队列“。

快速跟踪计划有助于开发,加速对严重条件的新药物的审查,这有可能解决未满足的医疗需求。目的是加快患者的新治疗方案的可用性。收到快速轨道指定的产品有资格获得与FDA更频繁的互动,加速批准,优先审查和滚动生物制剂申请(BLA)审查的潜在资格。

“这种快速轨道指定展示了稳压因子对溶酶的切削刃基因治疗计划的持续兴趣。亚搏全站app下载它补充了2016年和2017年FDA授予的罕见的小儿疾病和孤儿药物指标“添加赖戈纳州首席监管人员Marie Deneux“我们期待与原子能机构密切合作,努力开发Lys-Gm101作为治疗GM1神经性疾病的潜在第一药。”亚博登录首页

该试验是一种介入,多中心,单臂,两级自适应设计研究,评估携带人β-半乳糖苷酶基因(GBL1)的心脉内递送的重组腺相关病毒载体血清型RH.10(AAVRH.10)(NCT04273269)。临床试验包括安全阶段和确认的疗效阶段。该试验将注册16名患者,诊断美国和欧洲的景点的早期或晚期婴儿GM1神经病症。亚博登录首页可以找到更多信息www.clinicaltrials.gov。

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).

关于溶酶

Lysogene是一种基因治亚搏全站app下载疗公司,专注于治疗中枢神经系统(CNS)的孤儿疾病。该公司建立了一种独特的能力,使得能够将基因疗法传递给CNS以治疗CNS的溶酶体疾病和其他遗传疾病。与Sarepta Therapeutics,Inc.合作的MPS IIIA中的2/3阶段临床试验正在进行中。在GM1神经节病菌中的适应性临床试验正在进行中。亚博登录首页根据溶酶和Sarepta Therapeutics,Inc。之间签署的协议,Sarepta Therapeutics,Inc。将在美国和欧洲以外的市场举办Lys-Saf302的独家商业权利;和溶酶将在欧洲维持Lys-Saf302的商业专用性。Lysogene还与Satt Concectus进行了独家全球许可协议,用于治疗脆弱的X综合征的基因治疗候选者,该遗传疾病与自闭症有关。亚搏全站app下载www.astridbowlby.com

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, these forward-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
文本,法语版本应该取代。

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

Lysogene Covid-19更新

取得联系

如果你需要更多的帮助,请随时联系我们。

    回到顶部