Dyne Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for DYNE-251, a treatment for patients with Duchenne muscular dystrophy (DMD) who are eligible for exon 51 skipping.

Dyne Therapeutics 宣布，美国食品药品监督管理局 (FDA) 已授予 DYNE-251 突破性疗法认定，该药物用于治疗适合外显子 51 跳跃的杜氏肌营养不良症 (DMD) 患者。

DYNE-251 is an investigational therapy that combines a phosphorodiamidate morpholino oligomer (PMO) with a transferrin receptor-binding fragment. This combination is intended to support dystrophin production in muscle and the central nervous system, contributing to improved muscle function.

DYNE-251 是一种研究性疗法，它将磷酰二胺吗啉代寡聚物 (PMO) 与转铁蛋白受体结合片段结合。这种组合旨在支持肌肉和中枢神经系统中的抗肌萎缩蛋白生成，从而有助于改善肌肉功能。

Duchenne muscular dystrophy is a rare genetic disorder caused by mutations in the DMD gene, resulting in the lack of dystrophin protein. The condition primarily affects males, with around 12,000 cases in the U.S. and 16,000 in the EU. Symptoms often begin in early childhood and progressively worsen, leading to loss of mobility, respiratory difficulties, and cardiac complications.

杜氏肌营养不良症是一种由DMD基因突变引起的罕见遗传病，导致抗肌萎缩蛋白缺乏。该病主要影响男性，在美国约有12,000例，在欧盟约有16,000例。症状通常在儿童早期开始，并逐步恶化，导致行动能力丧失、呼吸困难和心脏并发症。

There remains a strong need for effective treatments that improve quality of life and slow disease progression..

仍然迫切需要能够改善生活质量和延缓疾病进展的有效治疗方法。

The designation is supported by clinical data from the ongoing DELIVER trial.

该指定得到了正在进行的DELIVER试验的临床数据的支持。

DYNE-251 is designed to enable the production of near full-length dystrophin, a protein vital for muscle function. The treatment has shown sustained functional improvement over an 18-month period in key clinical measures, including time to rise and stride velocity. This development represents a significant step in addressing the needs of DMD patients..

DYNE-251旨在实现近乎全长的肌营养蛋白的生产，这种蛋白对肌肉功能至关重要。该治疗在包括起身时间和步速在内的关键临床指标上，显示出在18个月内的持续功能性改善。这一进展代表了满足DMD患者需求的重要一步。

This marks the second Breakthrough Therapy Designation received by Dyne Therapeutics, following a similar designation earlier in the year for DYNE-101 in the treatment of myotonic dystrophy type 1. Breakthrough Therapy Designation is intended to accelerate the development and review of treatments that may offer substantial improvements over existing therapies for serious conditions.

这标志着Dyne Therapeutics公司继今年早些时候DYNE-101在治疗1型肌强直性营养不良症上获得类似认定后，再次获得了突破性疗法认定。突破性疗法认定旨在加速可能为严重疾病提供大幅改善的治疗方法的开发和审评进程。

The designation provides benefits such as enhanced guidance from the FDA, frequent communication regarding regulatory strategy, and eligibility for rolling and priority review, which may shorten the approval timeline..

该指定提供了诸如来自 FDA 的更多指导、关于监管策略的频繁沟通以及滚动和优先审查的资格等好处，这可能会缩短审批时间。

DYNE-251 has already received Fast Track, Orphan Drug, and Rare Paediatric Disease designations in the U.S., as well as Orphan Drug designation in the EU.

DYNE-251 已在美国获得快速通道、孤儿药和罕见儿科疾病资格认定，并在欧盟获得孤儿药资格认定。

The DELIVER trial is a global, double-blind, placebo-controlled Phase 1/2 study assessing the safety, tolerability, and efficacy of DYNE-251 in DMD patients amenable to exon 51 skipping. The selected dose for the registrational cohort is 20 mg/kg administered every four weeks. The primary endpoint is the change in dystrophin protein levels at six months, measured by Western blot..

DELIVER试验是一项全球性、双盲、安慰剂对照的1/2期研究，评估DYNE-251在适合外显子51跳跃的DMD患者中的安全性、耐受性和有效性。注册队列的选定剂量为每四周给予20 mg/kg。主要终点是通过蛋白质印迹法测量的六个月内抗肌萎缩蛋白水平的变化。

Dyne has completed enrolment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial, with data expected in late 2025. A Biologics License Application (BLA) for accelerated approval in the U.S. is planned for early 2026. The company also aims to pursue regulatory approvals outside the U.S..

Dyne已完成DELIVER试验的注册扩展队列中32名患者的入组，预计数据将在2025年底公布。公司计划于2026年初提交美国生物制品许可申请（BLA）以获得加速批准，并且还计划寻求美国以外的监管批准。