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.




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。。


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.




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