Dyne Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-101, an investigational treatment for myotonic dystrophy type 1 (DM1).

Dyne Therapeutics 宣布，美国食品药品监督管理局 (FDA) 已授予 DYNE-101（一种用于治疗 1 型肌强直性营养不良 (DM1) 的在研药物）突破性疗法认定。

DYNE-101 is a novel therapeutic under investigation in the global Phase 1/2 ACHIEVE trial. It is composed of an antisense oligonucleotide (ASO) linked to a fragment antibody targeting transferrin receptor 1 (TfR1), facilitating delivery to muscle and the central nervous system.

DYNE-101 是一种正在全球 1/2 期 ACHIEVE 试验中研究的新型疗法。它由一种反义寡核苷酸 (ASO) 与靶向转铁蛋白受体 1 (TfR1) 的片段抗体结合而成，有助于递送到肌肉和中枢神经系统。

It aims to reduce toxic DMPK RNA and correct the abnormal splicing process seen in DM1.

它旨在减少有毒的DMPK RNA并纠正DM1中出现的异常剪接过程。

DM1 is a rare, inherited condition affecting muscle and nervous system function. It is caused by a CTG trinucleotide repeat expansion in the DMPK gene, leading to the accumulation of toxic RNA in the cell nucleus. This disrupts normal protein splicing and results in a broad range of symptoms including muscle stiffness, weakness, fatigue, heart and respiratory issues, and cognitive difficulties. .

DM1是一种罕见的遗传性疾病，影响肌肉和神经系统的功能。它是由DMPK基因中的CTG三核苷酸重复扩增引起的，导致细胞核内有毒RNA的积累。这会破坏正常的蛋白质剪接，并引发广泛的症状，包括肌肉僵硬、无力、疲劳、心脏和呼吸问题以及认知困难。

There are currently no approved therapies that modify the course of the disease. DM1 is estimated to affect over 40,000 individuals in the U.S. and more than 74,000 in Europe.

目前尚无获批的可以改变疾病进程的疗法。据估计，DM1在美国影响超过40,000人，在欧洲影响超过74,000人。

The announcement follows a recent Type C meeting with the FDA and the availability of new long-term functional data. The company also shared its updated regulatory pathway, aiming for Accelerated Approval in the United States.

该公告是在最近与 FDA 举行的 Type C 会议以及新的长期功能性数据发布之后发布的。公司还分享了其更新的监管路径，目标是获得美国的加速批准。

Dyne plans to use these results, along with data from other parts of the ACHIEVE trial, to support a potential Accelerated Approval submission by the end of 2026. A confirmatory Phase 3 trial is planned to begin in the first quarter of 2026.

Dyne计划利用这些结果以及ACHIEVE试验其他部分的数据，以在2026年底前支持潜在的加速批准申请。一项确证性的第3阶段试验计划于2026年第一季度开始。

The company is also exploring accelerated regulatory pathways in other countries.

公司还在探索其他国家的快速监管途径。

The treatment continues to demonstrate a favourable safety profile, with no serious adverse events related to the drug reported in 56 participants.

该治疗继续显示出良好的安全性，56名参与者中未报告与药物相关的严重不良事件。

As of 31 March 2025, Dyne reported cash and investments totalling $677.5 million and expects to maintain operations into the fourth quarter of 2026.

截至2025年3月31日，Dyne报告现金和投资总额为6.775亿美元，并预计能够维持运营至2026年第四季度。

Breakthrough Therapy Designation is intended to speed up the development of medicines showing early evidence of significant improvement over existing therapies.

突破性疗法指定旨在加速显示出比现有疗法有显著改善的药物的开发。

Benefits include increased guidance from senior FDA officials, frequent communications on trial design and regulatory issues, and eligibility for expedited reviews.

好处包括来自FDA高级官员的更多指导、关于试验设计和监管问题的频繁沟通，以及获得快速审查的资格。