Lysogene宣布在MPS IIIA AAVance 2/3期临床试验LYS-SAF302最新

Lysogene宣布在MPS IIIA AAVance 2/3期临床试验LYS-SAF302最新150150Lysogene

法国巴黎 - 2020年6月5日在上午08:00 CEST- Lysogene(FR0013233475 - LYS), a Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced that, following discussions with the U.S. Food and Drug Administration (FDA), a clinical hold was issued for the clinical trial AAVance (NCT03612869), a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). To date, 19 out of 20 patients have been treated, and all patients remain in the study and are being followed per study protocol. The primary and secondary trial endpoints are based on the analyses of these 19 patients already enrolled and there is no anticipated impact on the current clinical trial timelines.

临床保持遵循在脑内注射部位在MRI图像局部发现有些患者的观察。调查结果的局部性质表明要在交货期电位连接。迄今为止,还没有临床症状已被观察到,可以直接归因于观察到的MRI表现,所有的调查结果已经上报主管部门,伦理委员会和审判的DSMB(数据安全监测委员会)。所有试验参与者继续通过他们的医疗团队进行密切监测。一个前进的道路已与FDA讨论,并Lysogene期待着收集更多信息,以更好地了解LYS-SAF302的安全性。

“我要感谢家人对他们参与研究”亚博福彩3d说过苏菲奥利弗博士,首席医疗官Lysogene。“我们考虑患者安全的最优先事项,并相信LYS-SAF302的临床开发可以提供给患者有意义的益处与这种破坏性疾病。现在,我们将专注于收集更多信息,并继续与FDA合作,以确定适当的前进道路“。

关于Lysogene

Lysogene是基因治疗公亚搏全站app下载司集中于中枢神经系统(CNS)的罕见疾病的治疗。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 registration document (Document de référence), 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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