WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1).

马萨诸塞州沃尔瑟姆，2025年1月21日（环球通讯社）--Dyne Therapeutics，Inc.（纳斯达克：DYN），一家临床阶段的神经肌肉疾病公司，专注于为患有遗传性疾病的人推进生命转化疗法，今天宣布，美国食品和药物管理局（FDA）已批准Dyne-101用于治疗强直性营养不良1型（DM1）的快速通道。

DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial..

DYNE-101目前正在进行的1/2期ACHIVE全球临床试验中进行评估。。

“This Fast Track designation comes on the heels of robust clinical data from our ACHIEVE trial, which demonstrated substantial functional benefit for patients across a range of clinical measures and a compelling effect on the key disease biomarker of splicing correction,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne.

达因首席医学官道格·克尔（DougKerr）医学博士说：“这一快速通道的命名是在我们的ACHAGE试验的强大临床数据之后进行的，该试验证明了在一系列临床措施中对患者的实质性功能益处，并对剪接矫正的关键疾病生物标志物产生了令人信服的影响。”。

“DM1 is a devastating disease with no approved therapies, and we are driven to deliver DYNE-101, a potentially transformative medicine, to patients as quickly as possible.”.

“DM1是一种毁灭性疾病，没有经过批准的治疗方法，我们必须尽快向患者提供DYNE-101，一种潜在的变革性药物。”。

The FDA grants Fast Track designation to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval.

FDA授予快速通道指定，以促进药物的开发和加速审查，以治疗严重疾病并满足未满足的医疗需求，目标是尽早向患者提供重要的新药。获得快速通道指定的药物可能有资格与FDA进行更频繁的会议和沟通，并对任何上市批准申请进行滚动审查。

Rolling review allows a company to submit completed sections of its Biologic License Application (BLA) for review by FDA as they are ready, rather than waiting until the entire application is complete for FDA review. This may lead to earlier drug approval and access by patients. A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met..

滚动审查允许公司在准备就绪时提交其生物许可证申请（BLA）的完整部分供FDA审查，而不是等到整个申请完成后再进行FDA审查。这可能会导致患者更早获得药物批准和使用。如果符合相关标准，接受快速通道指定的药物也可能有资格获得加速批准和优先审查。。

About DYNE-101

关于DYNE-101

DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with DM1. DYNE-101 consists of an antisense oligonucleotide (ASO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle.

DYNE-101是针对DM1患者的1/2期global ACHIEVE临床试验中正在评估的一种研究性治疗药物。DYNE-101由与片段抗体（Fab）偶联的反义寡核苷酸（ASO）组成，该片段抗体与在肌肉上高度表达的转铁蛋白受体1（TfR1）结合。

It is designed to enable targeted muscle tissue delivery with the goal of reducing toxic .

它旨在实现靶向肌肉组织输送，以减少毒性。

DMPK

DMPK

RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease progression. DYNE-101 has been granted orphan drug designation by the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of DM1..

细胞核中的RNA，释放剪接蛋白，允许正常的mRNA加工和正常蛋白的翻译，并可能阻止或逆转疾病进展。DYNE-101已被美国食品和药物管理局和欧洲药品管理局授予孤儿药称号，用于治疗DM1。。

About Myotonic Dystrophy Type 1 (DM1)

关于1型强直性营养不良（DM1）

DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. It is a monogenic, autosomal dominant disease caused by an abnormal trinucleotide expansion in a region of the

DM1是一种罕见的进行性遗传疾病，会影响骨骼肌，心肌和平滑肌。

DMPK

DMPK

gene. This expansion of CTG repeats causes toxic RNA to cluster in the nucleus, forming nuclear foci and altering the splicing of multiple proteins essential for normal cellular function. This altered splicing, or spliceopathy, results in a wide range of symptoms. People living with DM1 typically experience myotonia and progressive weakness of major muscle groups, which can affect mobility, breathing, heart function, speech, digestion and vision as well as cognition.

基因。CTG重复序列的这种扩增导致有毒RNA聚集在细胞核中，形成核灶并改变正常细胞功能所必需的多种蛋白质的剪接。这种改变的剪接或剪接病会导致多种症状。患有DM1的人通常会出现肌强直和主要肌肉群的进行性无力，这会影响活动能力，呼吸，心脏功能，言语，消化和视力以及认知。

DM1 is estimated to affect more than 40,000 people in the United States and over 74,000 people in Europe, but there are currently no approved disease-modifying therapies..

据估计，DM1在美国影响40000多人，在欧洲影响74000多人，但目前还没有批准的疾病缓解疗法。。

About Dyne Therapeutics

关于Dyne Therapeutics

Dyne Therapeutics is focused on discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that are designed to overcome limitations in delivery to muscle tissue and the central nervous system (CNS).

Dyne Therapeutics专注于为患有遗传驱动的神经肌肉疾病的人发现和推进创新的改变生活的疗法。利用其FORCE™平台的模块性，Dyne正在开发靶向治疗剂，旨在克服向肌肉组织和中枢神经系统（CNS）输送的局限性。

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

Dyne在神经肌肉疾病方面拥有广泛的渠道，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）和庞贝氏病的临床前计划。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。。