Lysogene在AAVance临床试验的患者MPS IIIA评估LYS-SAF302提供更新

Lysogene在AAVance临床试验的患者MPS IIIA评估LYS-SAF302提供更新150150Lysogene

法国巴黎 - 2020年10月15日08:00 AM - Lysogene(FR0013233475 - LYS),一期3基因治疗平台公司亚搏全站app下载针对中枢神经系统(CNS)疾病,今天宣布,病人已经在AAVance临床试验(NCT03612869),全球2/3期基因疗法试验为粘多糖病IIIA型治疗离世(MPS IIIA,也称为Sanfilippo综合症类型A)。MPS IIIA是一种严重的,危及生命,继承神经退行性溶酶体贮积病,特点是棘手的行为问题和发展回归导致早期死亡。

死亡的直接原因目前还不清楚,正在收集更多的信息。在这个时候,没有证据表明该事件被链接到研究药物施用。

Lysogene孜孜以下每个研究方案的18名患者谁已经在临床试验中接受治疗。

Lysogene是深受这个孩子的去世感到非常难过和最深切的同情到家庭。该公司仍然致力于LYS-SAF302发展计划和圣菲利波病人社区。该公司计划提供基于双方正在进行数据收集和未来的管理状态更新LYS-SAF302计划的进一步资料。

关于Lysogene

Lysogene是基因治疗公亚搏全站app下载司专注于中枢神经系统(CNS)的罕见疾病的治疗。本公司已经建立了一个独特的功能,实现安全和有效地提供基因治疗的CNS治疗溶酶体疾病和中枢神经系统的其他遗传性疾病。A相2/3临床试验在MPS IIIA与Sarepta治疗合伙公司正在进行并且在GM1神经节苷脂的相位1/3临床试验是在制备。亚博登录首页按照Lysogene和Sarepta Therapeutics公司,Sarepta治疗之间签订的协议,公司将持有的美国和欧洲以外市场的独家商业权利LYS-SAF302;和Lysogene将保持在欧洲LYS-SAF302的商业排他性。Lysogene也与学术合作伙伴合作,以定义脆性X综合征,遗传性疾病与治疗孤独症的发展战略。www.astridbowlby.com。

前瞻性声明

本新闻稿可能包含某些前瞻性陈述,特别是对公司的阶段进度2-3临床试验和现金跑道。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 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, 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.

本新闻稿中的法语和英语编写的。在两个文本之间的差异的情况下,法语版将取代。

往来

斯特凡杜兰特DES奥尔努瓦

首席财务官

stephane.durant-des-aulnois@lysogene.com

+ 33 1 41 43 03 99

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