WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced new preclinical data in a Pompe disease model demonstrating the potential of the FORCE™ platform to deliver enzyme replacement therapy to cardiac and skeletal muscle and the central nervous system (CNS).

马萨诸塞州沃尔瑟姆，2024年6月24日（环球通讯社）--Dyne Therapeutics，Inc.（纳斯达克：DYN），一家临床阶段肌肉疾病公司，专注于为患有遗传性疾病的人推进创新的生命转化疗法，今天宣布了庞贝病模型中的新临床前数据，展示了FORCE™平台向心脏和骨骼肌以及中枢神经系统（CNS）提供酶替代疗法的潜力。

The data were presented at the New Directions in Biology and Disease of Skeletal Muscle Conference, being held June 23-26 in Fort Lauderdale, FL..

这些数据发表在6月23日至26日在佛罗里达州劳德代尔堡举行的骨骼肌生物学和疾病新方向会议上。。

“These preclinical data show for the first time the ability of FORCE to deliver enzymes to skeletal and cardiac muscle as well as the CNS, expanding the modularity of our platform beyond oligonucleotides. The data support the potential of FORCE to enable effective enzyme replacement therapy with the opportunity to substantially improve upon available treatments for Pompe disease,” Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne.

。

“We look forward to continuing to explore this application of our platform as part of our mission to deliver life-transforming therapies for people with serious muscle diseases.”.

“我们期待着继续探索我们平台的这种应用，作为我们为严重肌肉疾病患者提供改变生活疗法的使命的一部分。”。

Pompe disease is a rare, severe neuromuscular disorder caused by deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA leads to glycogen accumulation and increase in lysosomal size in muscle and subsequent weakness, cardiomyopathy and respiratory failure. Enzyme replacement therapy with GAA is the standard of care and increases survival but has inadequate efficacy in skeletal muscle.

庞贝病是一种罕见的严重神经肌肉疾病，由溶酶体酶酸性α-葡萄糖苷酶（GAA）缺乏引起。缺乏GAA会导致糖原积累和肌肉溶酶体大小增加，并随后导致虚弱，心肌病和呼吸衰竭。用GAA进行酶替代疗法是护理的标准，可以提高生存率，但对骨骼肌的疗效不足。

Pompe is also characterized by CNS manifestations, including behavioral and cognitive deficits due to glycogen accumulation in CNS cells, which are not addressed by the standard of care therapy..

庞贝氏症还具有中枢神经系统表现的特征，包括由于中枢神经系统细胞中糖原积累而导致的行为和认知缺陷，而标准护理疗法并未解决这一问题。。

Dyne engineered FORCE-GAA by leveraging the FORCE platform and evaluated efficacy in vivo using hTfR1/6Neo mice, that were developed by crossing the well-established 6Neo mouse model of Pompe with mice expressing human transferrin receptor 1. Intravenous administration of FORCE-GAA cleared glycogen in muscle and the CNS and normalized lysosomal size in hTfR1/6Neo mice.

Dyne通过利用FORCE平台设计了FORCE-GAA，并使用hTfR1/6Neo小鼠评估了体内功效，该小鼠是通过将已建立的庞贝6Neo小鼠模型与表达人转铁蛋白受体1的小鼠杂交而开发的。静脉注射FORCE-GAA可清除肌肉和中枢神经系统中的糖原，并使hTfR1/6Neo小鼠的溶酶体大小正常化。

FORCE-GAA reduced serum neurofilament light chain, a biomarker of axonal injury, providing evidence of benefit in the CNS. FORCE-GAA also displayed superior efficacy and dose potency compared to GAA alone. Additional data with FORCE-GAA showed the potential for monthly dosing which is less frequent than approved enzyme replacement therapies..

FORCE-GAA减少了血清神经丝轻链，这是轴突损伤的生物标志物，为中枢神经系统提供了有益的证据。与单独的GAA相比，FORCE-GAA还显示出优异的功效和剂量效力。FORCE-GAA的其他数据显示，每月给药的可能性低于批准的酶替代疗法。。

The presentation entitled, “FORCE™ Platform for the Development of Targeted Therapeutics for Rare Muscle Diseases” is available in the Scientific Publications & Presentations section of Dyne’s website at https://www.dyne-tx.com/our-forcetm-publications/.

题为“FORCE™平台用于开发针对罕见肌肉疾病的靶向治疗方法”的演示文稿可在Dyne网站的科学出版物和演示文稿部分获得，网址为https://www.dyne-tx.com/our-forcetm-publications/.

About Pompe Disease

关于庞贝病

Pompe disease is a severe neuromuscular disorder caused by a deficiency of the lysosomal enzyme, acid alpha glucosidase (GAA). Lack of GAA causes glycogen accumulation in tissue leading to muscle weakness, cardiomyopathy, respiratory failure, and central nervous system (CNS) manifestations. Pompe disease belongs to a group of diseases known as the lysosomal storage disorders (LSDs). It can present as infantile-onset, the most severe form of the disease with early onset of symptoms in infancy that rapidly progress, or late-onset, which progressively damages muscles over time.

庞贝氏病是一种严重的神经肌肉疾病，由溶酶体酶酸性α-葡萄糖苷酶（GAA）缺乏引起。缺乏GAA会导致组织中糖原积累，导致肌肉无力，心肌病，呼吸衰竭和中枢神经系统（CNS）表现。庞贝病属于一组称为溶酶体贮积病（LSD）的疾病。它可以表现为婴儿期发作，这是该疾病最严重的形式，在婴儿期症状发作较早，进展迅速，或发作较晚，随着时间的推移逐渐损害肌肉。

An estimated 5,000 to 10,000 individuals worldwide are affected by Pompe. .

。。

About the FORCE™ Platform

关于FORCE™平台

The proprietary FORCE™ platform drives Dyne’s efforts to develop targeted, modern oligonucleotide therapeutics with the potential to be life-transforming for patients with serious muscle diseases. Dyne designed the FORCE platform using its deep knowledge of muscle biology and oligonucleotide therapeutics to overcome the current limitations in delivery to muscle tissue with the goal of stopping or reversing disease progression.

专有的FORCE™平台推动了Dyne开发有针对性的现代寡核苷酸疗法的努力，这些疗法有可能改变严重肌肉疾病患者的生活。达因利用其对肌肉生物学和寡核苷酸治疗学的深入知识设计了FORCE平台，以克服目前向肌肉组织输送的局限性，从而阻止或逆转疾病进展。

The FORCE platform leverages the importance of transferrin receptor 1 (TfR1) in muscle biology as the foundation for its novel approach. TfR1, which is highly expressed on the surface of muscle cells, is required for iron transport into muscle cells. Dyne links therapeutic payloads to its TfR1-binding fragment antibody (Fab) to develop targeted therapeutics for muscle diseases. .

FORCE平台利用转铁蛋白受体1（TfR1）在肌肉生物学中的重要性作为其新方法的基础。TfR1在肌肉细胞表面高度表达，是铁转运到肌肉细胞所必需的。达因将治疗有效载荷与其TfR1结合片段抗体（Fab）联系起来，以开发针对肌肉疾病的靶向治疗方法。。

About Dyne Therapeutics

关于Dyne Therapeutics

Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue.

Dyne Therapeutics是一家临床阶段的肌肉疾病公司，专注于为患有遗传性疾病的人推进创新的改变生活的疗法。凭借其专有的FORCE™平台，Dyne正在开发现代寡核苷酸疗法，旨在克服向肌肉组织输送的限制。

Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit https://www.dyne-tx.com, and follow us on X, LinkedIn and Facebook..

Dyne拥有治疗严重肌肉疾病的广泛渠道，包括1型强直性肌营养不良症（DM1）和杜兴氏肌营养不良症（DMD）的临床计划以及面肩肱型肌营养不良症（FSHD）的临床前计划。有关更多信息，请访问https://www.dyne-tx.com，并在X、LinkedIn和Facebook上关注我们。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the anticipated timelines for reporting additional data for FORCE-GAA, expectations regarding the initiation of additional preclinical studies or clinical trials of FORCE-GAA, and plans to provide future updates on pipeline programs, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995.

本新闻稿包含前瞻性声明，涉及重大风险和不确定性。本新闻稿中包含的除历史事实声明外的所有声明，包括有关Dyne战略、未来运营、前景和计划、管理目标、FORCE平台潜力、FORCE-GAA额外数据报告的预期时间表、关于启动FORCE-GAA额外临床前研究或临床试验的期望以及提供管道计划未来更新的计划的声明，均构成1995年《私人证券诉讼改革法》意义下的前瞻性声明。

The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

“预期”，“相信”，“继续”，“可能”，“估计”，“预期”，“打算”，“可能”，“可能”，“目标”，“正在进行”，“计划”，“预测”，“项目”，“潜在”，“应该”或“会”，或这些术语的否定词或其他类似术语旨在识别前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。

Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne’s ability to enroll patients in clini.

Dyne可能无法实际实现这些前瞻性声明中披露的计划、意图或期望，您不应过度依赖这些前瞻性声明。由于各种重要因素，实际结果或事件可能与这些前瞻性声明中披露的计划、意图和期望存在重大差异，包括：候选产品的识别和开发固有的不确定性，包括临床前研究和临床试验的开始和完成；临床前研究和临床试验结果的可用性和时间不确定性；以及达因在clini中招募患者的时间和能力。

Contacts:

联系人：

Investors

投资者

Amy Reilly

艾米·赖利

areilly@dyne-tx.com

areilly@dyne-tx.com

857-341-1203

857-341-1203

Media

媒体

Stacy Nartker

Stacy Nartker。

snartker@dyne-tx.com

snartker@dyne-tx.com

781-317-1938

781-317-1938