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Stoke Therapeutics to Host Webcast to Discuss Successful Global Regulatory Alignment for a Phase 3 Study of Zorevunersen as Potentially the First Disease Modifying Medicine for Dravet Syndrome

Stoke Therapeutics to Host Webcast to Discuss Successful Global Regulatory Alignment for a Phase 3 Study of Zorevunersen as Potentially the First Disease Modifying Medicine for Dravet Syndrome

Stoke Therapeutics将举办网络广播,讨论针对Zorevunersen的第三阶段研究的成功全球货币监管对齐,该药物有可能成为首个可改变疾病进程的药物,用于Dravet综合症。
Stoke Therapeutics ·  01/06 13:00

BEDFORD, Mass.--(BUSINESS WIRE)--Jan. 6, 2025--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, today announced that its management team will host a webcast and conference call for investors and analysts to discuss successful alignment with global regulatory agencies related to a Phase 3 study of zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet syndrome.

美国马萨诸塞州贝德福德--(商业通讯)--2025年1月6日--Stoke Therapeutics, Inc.(纳斯达克:STOK)是一家致力于通过利用RNA药物恢复蛋白质表达的生物技术公司,今天宣布其管理团队将为投资者和分析师举办网络广播和电话会议,讨论与全球监管机构在zorevunersen的三期研究中成功对接的情况,这可能是治疗Dravet综合症的第一种疾病修正药物。

The webcast will be conducted Tuesday, January 7, 2025 at 8:00am Eastern Time and can be accessed from the Investors & News section of Stoke's website at . Research analysts who plan to join the call and participate in the Q&A session may register here to receive the dial-in details and a unique PIN. For all others, the listen-only webcast can be accessed by clicking here. A replay of the webcast will be archived and available for at least 90 days following the event.

网络广播将于2025年1月7日星期二东部时间上午8:00进行,投资者可以通过访问Stoke网站的投资者和新闻部分来获取。计划参加电话会议和Q&A环节的研究分析师可以注册以获取拨入详情和唯一PIN码。对于其他人,可以通过点击这里收听仅供听众使用的网络广播。网络广播的录音将在活动结束后至少保存90天。

About Stoke Therapeutics

关于Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Stoke's first compound, zorevunersen (STK-001), is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease. Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke's initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting its belief in the broad potential for its proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit .

Stoke Therapeutics(纳斯达克:STOK)是一家致力于通过利用RNA药物恢复蛋白质表达的生物技术公司。利用Stoke独特的TANGO(核基因输出的靶向增强)方法,Stoke正在开发反义寡核苷酸(ASOs)以有选择性地恢复蛋白质水平。Stoke的第一个化合物zorevunersen(STk-001)正在进行临床测试,治疗Dravet综合症,这是一种严重且进展迅速的遗传性癫痫。Dravet综合症是由半数不足引起的许多疾病之一,其中正常蛋白水平丧失约50%会导致疾病。Stoke正在开发STk-002,用于治疗常染色体显性视神经萎缩(ADOA),这是最常见的遗传性视神经疾病。Stoke最初关注于半数不足和中枢神经系统及眼睛的疾病,尽管在其他器官、组织和系统中已证明了概念验证,支持其对其专有方法广泛潜力的信念。Stoke总部位于马萨诸塞州贝德福德,并在马萨诸塞州剑桥设有办事处。有关更多信息,请访问。

About Zorevunersen

关于Zorevunersen

Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that zorevunersen, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. Zorevunersen is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Zorevunersen has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene.

Zorevunersen是一种用于治疗Dravet综合症的新研究药物,目前正在进行临床试验。Stoke认为,zorevunersen作为一种专有的反义寡核苷酸(ASO),有潜力成为第一个能够针对Dravet综合症遗传原因的病程改变疗法。Zorevunersen旨在通过利用SCN1A基因的非突变(野生型)副本上调NaV1.1蛋白的表达,以恢复生理NaV1.1水平,从而减少癫痫发作的发生以及显著的非癫痫共病症。Zorevunersen已获得FDA和EMA的孤儿药认证。FDA还授予zorevunersen稀有小儿疾病认证和突破疗法认证,以治疗具有确认突变的Dravet综合症,且不与SCN1A基因的功能获得相关。

View source version on businesswire.com:

在businesswire.com查看源版本:

Stoke Media & Investor Contacts:
Dawn Kalmar
Chief Communications Officer
dkalmar@stoketherapeutics.com
781-303-8302
Doug Snow
Director, Communications & Investor Relations
IR@stoketherapeutics.com
508-642-6485

Stoke媒体与投资者联系方式:
道恩·卡尔马尔
首席通讯官
dkalmar@stoketherapeutics.com
781-303-8302
道格·斯诺
董事,通信-半导体和投资者关系
IR@stoketherapeutics.com
508-642-6485

Source: Stoke Therapeutics, Inc.

来源:Stoke Therapeutics, Inc.

译文内容由第三方软件翻译。


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