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Stealth BioTherapeutics Announces Oral Presentation of New Duchenne Muscular Dystrophy Data at the 2022 Muscular Dystrophy Association Clinical and Scientific Conference

Stealth BioTherapeutics Announces Oral Presentation of New Duchenne Muscular Dystrophy Data at the 2022 Muscular Dystrophy Association Clinical and Scientific Conference

隱形生物治療公司宣佈在2022年肌營養不良症協會臨牀和科學會議上口頭公佈新的Duchenne肌營養不良症數據
PR Newswire ·  2022/02/08 20:46

BOSTON, Feb. 8, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the company will have an oral presentation at the upcoming 2022 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, being held March 13-16, 2022 in Nashville, Tennessee. The presentation showcases data demonstrating a potential relationship between elamipretide and an exon skipping phosphorodiamidate morpholino oligomer (PMO) in the X-linked muscular dystrophy (mdx) mouse model.  PMOs are one of the few approved drug classes for the treatment of patients with Duchenne muscular dystrophy (DMD).

波士頓,2022年2月8日/美通社/--隱形生物治療公司(納斯達克代碼:MITO)是一家臨牀階段生物技術公司,專注於發現、開發和商業化治療涉及線粒體功能障礙的疾病的新療法。該公司今天宣佈,該公司將在即將於2022年3月13日至16日舉行的2022年肌營養不良症協會臨牀和科學大會上作口頭陳述。田納西州納什維爾。該報告展示了在X-連鎖肌營養不良症(MDX)小鼠模型中,伊拉米肽和一種跳過外顯子的磷二酰嗎啉低聚物(PMO)之間的潛在關係的數據。PMOS是為數不多的被批准用於治療Duchenne肌營養不良症(DMD)患者的藥物類別之一。

The presentation details are as follows:

介紹詳情如下:

Title: The mitochondria-targeting peptide elamipretide potentiates dystrophin expression induced by an exon-skipping morpholino in the mdx mouse model
Presenter: David A. Brown, Ph.D.
Date: Wednesday, March 16, 2022
Time: 9:10-9:25 am CST
Location: Gaylord Opryland Resort & Convention Center Hermitage AB Room

標題線粒體靶向多肽elamipretide增強mdx小鼠模型中外顯子跳過嗎啡肽誘導的dystrophin表達
演示者作者:David A.Brown,Ph.D.
日期: 2022年3月16日星期三
時間中部夏令時上午9:10-9:25
位置:蓋洛德·奧普里蘭度假村及會議中心赫米蒂奇AB房

"We are encouraged to learn that administration of elamipretide in addition to standard-of-care therapy may amplify therapeutic benefit in this model of Duchenne muscular dystrophy," said Reenie McCarthy, Chief Executive Officer of Stealth.  "With this finding, we hope to commence additional clinical development efforts with an ultimate goal of improving the standard of care for individuals suffering from the neuromuscular, as well as the cardiomyopathic, symptoms of this devastating and life-limiting disease."

Stealth公司首席執行官Reenie McCarthy説:“我們感到鼓舞的是,在標準治療的基礎上加用伊拉米肽,可能會擴大這種Duchenne肌營養不良症模型的治療效果。”有了這一發現,我們希望開始更多的臨牀開發工作,最終目標是提高患有這種毀滅性和限制生命的疾病的神經肌肉和心肌疾病症狀的個人的護理標準。“

About Stealth

關於隱形行動

We are a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the eye, the neuromuscular system, the heart and the brain. We believe our lead product candidate, elamipretide, has the potential to treat ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration, rare neuromuscular disorders, such as primary mitochondrial myopathy caused by nuclear DNA mutations and DMD, and rare cardiomyopathies, such as Barth syndrome. We are evaluating our second-generation clinical-stage candidate, SBT-272, for rare neurological disease indications, such as amyotrophic lateral sclerosis and frontotemporal dementia, following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeted compounds which may be nominated as therapeutic product candidates or utilized as mitochondria-targeted vectors to deliver other compounds to mitochondria.

我們是一家臨牀階段的生物技術公司,專注於發現、開發和商業化治療涉及線粒體功能障礙的疾病的新療法。線粒體幾乎存在於人體的每一個細胞中,是人體產生能量的主要來源,對正常器官功能至關重要。線粒體功能障礙是許多罕見的遺傳性疾病的特徵,並與許多常見的與年齡相關的疾病有關,通常涉及眼睛、神經肌肉系統、心臟和大腦等能量需求較高的器官系統。我們相信,我們的主要候選產品elamipretie有潛力治療導致線粒體功能障礙的眼科疾病,如干性老年性黃斑變性,罕見的神經肌肉疾病,如由核DNA突變和DMD引起的原發性線粒體肌病,以及罕見的心肌疾病,如Barth綜合徵。根據有希望的臨牀前數據,我們正在評估我們的第二代臨牀階段候選藥物SBT-272,用於治療罕見的神經疾病適應症,如肌萎縮側索硬化症和額顳葉痴呆。我們優化了我們的發現平臺,以確定新的線粒體靶向化合物,這些化合物可能被提名為候選治療產品,或者被用作線粒體靶向載體,將其他化合物運送到線粒體。

Investor Relations
Stern Investor Relations
Janhavi Mohite, 212-362-1200
[email protected] 

投資者關係
斯特恩投資者關係
Janhavi Mohite,212-362-1200
[受電子郵件保護]

SOURCE Stealth BioTherapeutics Inc.

來源:隱形生物治療公司。

譯文內容由第三人軟體翻譯。


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