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 (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 .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.
美國馬薩諸塞州貝德福德--（商業通訊）--2025年1月6日--Stoke Therapeutics, Inc.（納斯達克：STOK）是一家致力於通過利用RNA藥物恢復蛋白質表達的生物技術公司，今天宣佈其管理團隊將爲投資者和分析師舉辦網絡廣播和電話會議，討論與全球監管機構在zorevunersen的三期研究中成功對接的情況，這可能是治療Dravet綜合症的第一種疾病修正藥物。
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.
網絡廣播將於2025年1月7日星期二東部時間上午8:00進行，投資者可以通過訪問Stoke網站的投資者和新聞部分來獲取。計劃參加電話會議和Q&A環節的研究分析師可以註冊以獲取撥入詳情和唯一PIN碼。對於其他人，可以通過點擊這裏收聽僅供聽衆使用的網絡廣播。網絡廣播的錄音將在活動結束後至少保存90天。
About Stoke Therapeutics
關於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（納斯達克：STOK）是一家致力於通過利用RNA藥物恢復蛋白質表達的生物技術公司。利用Stoke獨特的TANGO（核基因輸出的靶向增強）方法，Stoke正在開發反義寡核苷酸（ASOS）以有選擇性地恢復蛋白質水平。Stoke的第一個化合物zorevunersen（STk-001）正在進行臨牀測試，治療Dravet綜合症，這是一種嚴重且進展迅速的遺傳性癲癇。Dravet綜合症是由半數不足引起的許多疾病之一，其中正常蛋白水平喪失約50%會導致疾病。Stoke正在開發STk-002，用於治療常染色體顯性視神經萎縮（ADOA），這是最常見的遺傳性視神經疾病。Stoke最初關注於半數不足和中樞神經系統及眼睛的疾病，儘管在其他器官、組織和系統中已證明了概念驗證，支持其對其專有方法廣泛潛力的信念。Stoke總部位於馬薩諸塞州貝德福德，並在馬薩諸塞州劍橋設有辦事處。有關更多信息，請訪問。
About Zorevunersen
關於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是一種用於治療Dravet綜合症的新研究藥物，目前正在進行臨牀試驗。Stoke認爲，zorevunersen作爲一種專有的反義寡核苷酸（ASO），有潛力成爲第一個能夠針對Dravet綜合症遺傳原因的病程改變療法。Zorevunersen旨在通過利用SCN1A基因的非突變（野生型）副本上調NaV1.1蛋白的表達，以恢復生理NaV1.1水平，從而減少癲癇發作的發生以及顯著的非癲癇共病症。Zorevunersen已獲得FDA和EMA的孤兒藥認證。FDA還授予zorevunersen稀有小兒疾病認證和突破療法認證，以治療具有確認突變的Dravet綜合症，且不與SCN1A基因的功能獲得相關。
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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媒體與投資者聯繫方式：
道恩·卡爾馬爾
首席通訊官
dkalmar@stoketherapeutics.com
781-303-8302
道格·斯諾
董事，通信和投資者關係
IR@stoketherapeutics.com
508-642-6485
Source: Stoke Therapeutics, Inc.
來源：Stoke Therapeutics, Inc.
譯文內容由第三人軟體翻譯。