A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).

开始日期2022-08-12

申办/合作机构

Dyne Therapeutics, Inc.

100 项与泽雷西孟特・罗司地生相关的临床结果

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100 项与泽雷西孟特・罗司地生相关的转化医学

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100 项与泽雷西孟特・罗司地生相关的专利（医药）

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项与泽雷西孟特・罗司地生相关的文献（医药）

2026-12-31·mAbs

Antibodies to watch in 2026

Review

作者: Pinto, Kieran ; Kapoor, Vaishali ; Kaplon, Hélène ; Crescioli, Silvia ; Reichert, Janice M. ; Chenoweth, Alicia ; Hsu, Yu-Shin

The Antibodies to Watch article series provides annual updates on commercial late-stage clinical development, regulatory review, and marketing approvals of antibody therapeutics. Since the first article was published in 2010, the late-stage pipeline has grown from 26 antibody therapeutics to over 200, while during the same time numerous molecules in late-stage studies either transitioned to regulatory review and were approved or were terminated. In this installment of the series, we recap first marketing approvals granted to 19 antibody therapeutics in 2025, discuss 26 molecules currently in regulatory review, including the bispecific antibody-drug conjugate izalontamab brengitecan, and predict which molecules of the 209 currently in the commercial late-stage pipeline might transition to regulatory review by the end of 2026. Most antibody therapeutics in the latter category are for non-cancer indications (16/21, 76%) and have a conventional format (13/21, 62%), but the category also includes numerous antibody-oligo or -drug conjugates, such as delpacibart etedesiran, delpacibart zotadirsen, zeleciment rostudirsen, sonesitatug vedotin, trastuzumab pamirtecan, and ifinatamab deruxtecan, as well as the bispecific petosemtamab. As antibody therapeutics development is a global enterprise, we also discuss trends in annual first approvals granted to antibody therapeutics in any country since 2010, stratified by the antibody's country of origin, documenting the notable increases in the total number of first approvals and those approved first in China. Finally, to benchmark the time typically required for clinical development and regulatory review, we calculated this period for recently approved antibody therapeutic products stratified by their therapeutic area, mechanism of action, format, and country of origin. Our data show that the development and approval period were typically ~6 years, but on average this period was shorter for China-originated products.

175

项与泽雷西孟特・罗司地生相关的新闻（医药）

2026-05-27

·BioSpace

FDA accepts BridgeBio’s application for potential first limb-girdle muscular dystrophy drug

iStock, Yutthana Gaetgeaw The FDA’s priority review acceptance of BridgeBio’s BBP-418 is another step toward what William Blair previously dubbed a “diversified commercial portfolio.” It also adds to the rapidly building momentum in muscular dystrophy more broadly. The FDA has accepted BridgeBio’s BBP-418 for priority review, setting up what William Blair analysts called the first of three potential blockbuster launches—and possibly paving the way for the first approved therapy for a rare type of muscular dystrophy. The FDA has set a target action date of November 27 for BBP-418’s new drug application in limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) and is not planning to hold an advisory committee meeting to discuss the application, BridgeBio announced on Wednesday, representing “incremental positives for an approval and launch of the product in late 2026 or early 2027,” William Blair said in a note to investors. BridgeBio shares rose 3% premarket Wednesday in response to the news, according to the firm, but were back down to $65.96 at the time of publication. BridgeBio is supporting BBP-418’s application with data from the Phase 3 FORTIFY trial, in which the oral therapy met all primary and secondary endpoints at the pre-specified 12-month interim analysis, according to the biotech’s announcement, “showing treated individuals improving while placebo recipients declined across every key measure.” Patients also saw increases in ambulatory and pulmonary function, and a 5% increase in predicted pulmonary volume versus placebo, BridgeBio reported in October 2025. Jefferies analysts at the time said the data represent a “best-case” scenario, adding that BBP-418 compares favorably with Sarepta Therapeutics’ SRP-9003, an investigational gene therapy for LGMD. That asset is currently in a Phase 3 trial. Neuroscience BridgeBio Pops on Late-Stage Limb-Girdle Muscular Dystrophy Data New interim data from a Phase III trial puts the company on track to FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy. October 27, 2025 · 2 min read · Dan Samorodnitsky Read more LGMD2I/R9 occurs when mutations in the FKRP protein diminish but do not totally abolish its function, resulting in skeletal muscle damage, first to the lower and then the upper limbs. Pulmonary muscle and cardiac muscle involvement occur later in the disease process, and “patients ultimately die from respiratory failure,” BridgeBio Neuromuscular CEO Christine Siu said in a prepared statement on Wednesday. “Data from the Phase III FORTIFY study have painted an impressive picture of the efficacy and safety of BBP-418, with consistent, highly statistically significant functional and biomarker efficacy over placebo in LGMD2I/R9 patients,” William Blair said on Wednesday. The analysts estimated worldwide sales of $1.1 billion in 2035 for the program. In a note on Wednesday, Jefferies analysts declared “90%+ confidence” in full approval of BBP-418 based on the FORTIFY interim results. “Net-net, $1B+ peak sales seems reasonable,” the firm added, modeling $600M+ in the U.S. For BridgeBio, the priority review of BBP-418 is just one step on the company’s ladder toward what William Blair in a May 12 note called a “diversified commercial portfolio.” That note referenced the NDA submission that day of encaleret for autosomal dominant hypocalcemia type 1—another possible blockbuster, according to the analysts. A third potential blockbuster, William Blair said, is infigratinib in hypochondroplasia, for which BridgeBio is on track to submit an NDA in the third quarter of this year, per a May 8 note. Muscular dystrophy momentum BridgeBio’s news adds to the rapidly building momentum across a range of muscular dystrophies. Duchenne muscular dystrophy, in particular, has seen positive news of late. Dyne Therapeutics submitted a Biologics License Application to the FDA earlier this week for its exon 51 skipper DYNE-251, while REGENXBIO, Wave Life Sciences and Capricor Therapeutics are also all nearing the regulatory finish line in DMD. Duchenne muscular dystrophy REGENXBIO, Novartis, Dyne near FDA submissions in muscular dystrophies Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line. May 26, 2026 · 9 min read · Heather McKenzie Read more Meanwhile, Novartis will soon learn whether its $12 billion buyout of Avidity last October was worth the risk. The company is anticipating data from a Phase 3 trial of the DMPK-targeting therapy del-desiran in 159 patients with myotonic dystrophy type 1 in the second half of this year. When asked by BioSpace whether this readout will make or break the acquisition, Oppenheimer’s Kostas Biliouris said “Mostly, yes. . . . This is perhaps 80% of the acquisition.”

临床3期并购申请上市优先审批临床结果

2026-05-26

·药明康德

从治疗DMD到长效降血脂，核酸药物正在跨过哪道坎？

近年来，寡核苷酸及相关核酸药物正在从少数罕见病和肝脏适应症，走向更广阔的疾病治疗场景。推动这一变化的，不只是序列设计、化学修饰或靶点生物学的进步，更是递送技术的持续突破。能否把治疗分子精准、高效地带到目标组织，正在成为决定这类药物能否进入更多疾病领域的关键因素。日前，礼来（Eli Lilly and Company）公布其PCSK9体内碱基编辑疗法VERVE-102的1b期Heart-2研究数据。该疗法通过GalNAc偶联脂质纳米颗粒（LNP）递送编码腺嘌呤碱基编辑器的mRNA和靶向PCSK9的引导RNA（gRNA），旨在通过一次给药实现对肝脏PCSK9基因的持久关闭。今日，Dyne Therapeutics宣布向美国FDA递交zeleciment rostudirsen（z-rostudirsen，也称DYNE-251）的生物制品许可申请（BLA），用于治疗适合外显子51跳跃的杜氏肌营养不良症（DMD）患者。该疗法则采用磷酰二胺吗啉寡聚体（PMO）与靶向转铁蛋白受体1（TfR1）的抗体片段偶联，以提升药物向肌肉和中枢神经系统相关组织递送的能力。这两款疗法所针对的疾病不同，技术路径也不同：一个探索通过长期降脂管理心血管疾病风险，另一个治疗严重遗传性神经肌肉疾病；一个探索体内编辑工具的肝脏靶向递送，另一个通过抗体片段偶联技术推动PMO在肌肉组织中的递送。但它们共同指向了一个重要趋势：递送技术正在决定核酸药物能够抵达哪里，也正在决定它们能够治疗哪些疾病。不只是“设计序列”，更要“抵达组织” 寡核苷酸药物的优势，在于它们可以直接作用于遗传信息或RNA层面，通过沉默、剪接调控、编辑或调节基因表达等方式，干预传统小分子和抗体药物难以触达的疾病机制。反义寡核苷酸（ASO）、siRNA、PMO、gRNA等不同类型分子，已经在罕见病、代谢疾病、神经系统疾病和心血管疾病等领域展现潜力。但这类药物也面临多重挑战。许多寡核苷酸分子带有较强亲水性和负电荷，难以像传统小分子那样自由穿越细胞膜；同时，它们还面临体内稳定性、组织分布、细胞摄取和安全性等多重限制。因此，对核酸药物而言，递送系统并不是附属技术，而是药物本身能否发挥作用的核心组成部分。过去，肝脏是核酸药物最成熟的靶器官之一。GalNAc偶联和LNP等技术，推动了siRNA、部分反义寡核苷酸和体内编辑疗法在肝脏相关疾病中的发展。如今，行业的下一个重要方向，是让核酸药物走向更难触达的组织，包括肌肉、中枢神经系统、肺部、眼部、免疫细胞和肿瘤微环境等。 VERVE-102：肝脏靶向递送支持“一次性治疗”的长期愿景礼来公布的VERVE-102数据，为肝脏靶向递送技术提供了一个具有代表性的案例。VERVE-102是一款在研体内碱基编辑药物，设计目标是在肝脏中关闭PCSK9基因，从而持久降低低密度脂蛋白胆固醇（LDL-C）。其治疗性载荷包括编码腺嘌呤碱基编辑器的mRNA和靶向PCSK9的gRNA，二者被封装在LNP中，并采用GalNAc偶联LNP递送技术，使LNP能够通过低密度脂蛋白受体（LDLR）或去唾液酸糖蛋白受体（ASGPR）进入肝细胞。这一设计的核心是尝试模拟天然存在的PCSK9功能缺失变异所带来的保护效应。遗传学研究提示，PCSK9长期处于较低水平的人群往往具有更低的LDL-C水平和更低的心血管疾病风险。基于这一生物学基础，VERVE-102希望通过一次体内编辑，让患者获得更加持久的降脂效果。在1b期Heart-2研究中，VERVE-102在不同剂量水平下显示出剂量依赖性的PCSK9和LDL-C水平下降。礼来的新闻稿指出，在最高剂量组，单次给药后PCSK9平均下降88%，LDL-C平均下降62%，且部分受试者随访时间最长达到18个月，仍观察到持久疗效。对于需要长期控制LDL-C的高危心血管疾病患者而言，这类治疗模式具有将部分慢病管理从长期、反复给药转向更持久甚至一次性干预的潜力。目前VERVE-102仍处于早期临床阶段。未来其疗效持久性和安全性仍需要长期随访结果以及更大规模临床研究进一步验证。但它已经提示了一个重要方向：当肝脏靶向递送平台足够成熟，核酸药物和体内编辑工具就有机会进入慢病长期管理领域，并重塑一些疾病的治疗模式。 Z-rostudirsen：PMO偶联递送进入DMD申报阶段如果说VERVE-102体现的是肝脏递送平台在慢病中的潜力，那么Dyne Therapeutics的z-rostudirsen则展示了另一个方向：通过偶联递送技术，提高寡核苷酸药物向肌肉等肝外组织递送的能力。 DMD是一种严重的X连锁进行性神经肌肉疾病，由DMD基因突变导致抗肌萎缩蛋白缺失或接近完全缺失。外显子跳跃疗法是DMD药物研发中的重要方向之一，其基本思路是通过调控前体mRNA剪接，使细胞产生截短但具有一定功能的抗肌萎缩蛋白。PMO是这一领域常用的寡核苷酸化学类型之一。 Z-rostudirsen由PMO与靶向TfR1的抗体片段偶联而成。TfR1在肌肉组织中具有较高表达，因此这一设计希望借助受体介导的递送机制，提高PMO进入相关组织和细胞的能力，并促进外显子51跳跃，从而产生近全长抗肌萎缩蛋白。Dyne此次递交BLA，申请用于治疗适合外显子51跳跃的DMD患者。这一案例的意义，不仅是一款DMD候选疗法的监管进展，更体现了寡核苷酸药物向肝外组织递送的一个重要方向：通过抗体、抗体片段、多肽、配体或其他偶联策略，让寡核苷酸药物更有效地进入目标组织。对于DMD这类全身性遗传病而言，递送效率不仅关系到分子能否进入肌肉组织，也关系到药物是否有机会在功能性临床终点、给药便利性和长期治疗负担之间取得更好的平衡。递送技术正在改变寡核苷酸药物开发从VERVE-102和z-rostudirsen可以看到，递送技术正在改变寡核苷酸及相关核酸药物的开发。首先，递送技术正在扩展可触达的靶组织范围。肝脏仍是核酸药物最成熟的应用阵地之一，但行业已经不满足于此。肌肉、中枢神经系统、眼部、肺部和免疫系统等组织，正在成为下一阶段递送创新的重要方向。不同组织的屏障结构、受体表达、细胞类型和疾病机制不同，意味着递送策略必须更加定制化。GalNAc、LNP、脂质偶联、多肽偶联、抗体或抗体片段偶联，以及其他受体介导递送方式，正在构成越来越丰富的技术工具箱。其次，递送技术正在拓展适应症范围。过去，许多寡核苷酸药物首先在罕见病中取得突破，因为这些疾病往往具有明确的遗传病因。随着递送效率和组织选择性的提升，核酸药物正在向更广泛的疾病领域延伸。VERVE-102代表着心血管疾病中长期干预的探索，z-rostudirsen代表着在神经肌肉遗传病中提高组织递送和功能转化的努力。未来，递送技术越成熟，核酸药物进入常见病和多基因疾病的可能性就越大。然而，复杂的递送技术也在抬高了药物开发和生产的门槛。寡核苷酸本身的序列设计、化学修饰、杂质控制、纯化方法、连接子、配体、偶联工艺、制剂开发、分析方法和质量标准，都会影响最终药物的安全性、有效性和可制造性。对于采用偶联或递送系统的寡核苷酸药物而言，开发难点已经不再停留在“把一条序列做出来”，而是要在分子设计、递送策略、工艺开发、分析表征和放大生产之间建立连续闭环。这也意味着，递送创新越向前推进，对开发平台的系统能力要求就越高。递送时代，需要一体化的寡核苷酸开发平台在寡核苷酸药物研发快速发展的背景下，从序列设计到生产制造的一体化能力对于提升研发效率与成功率至关重要。药明康德依托多业务平台协同优势，构建了覆盖药物发现、机制研究、药代动力学评价以及开发与生产的一体化寡核苷酸研发体系，为合作伙伴提供端到端支持。药明康德生物学业务平台（WuXi Biology）建立了覆盖设计、筛选、评价与转化研究的一体化寡核苷酸药物发现平台，可为合作伙伴提供端到端的寡核苷酸研发支持。平台依托自主建立的in silico序列设计体系，综合考虑脱靶风险、跨物种活性及序列特征等多维参数，实现候选寡核苷酸的高效设计与优选；结合超过1500种细胞资源及多类型细胞模型开展高通量体外筛选与功能评估，快速识别具有高敲降效率和良好特异性的候选分子。在安全性与机制研究方面，平台整合RNA转录组分析、免疫原性与细胞毒性检测，实现对脱靶效应与潜在安全风险的系统评估。在递送技术方面，除肝靶向GalNAc策略外，平台还支持肝外递送工具的发现，包括抗体和多肽等配体，并通过优化偶联位点与DAR提升递送效率。通过跨学科整合与标准化流程，WuXi Biology将寡核苷酸设计与优化、递送、安全性评估及药效评价紧密衔接，有效降低早期研发风险，加速创新寡核苷酸疗法迈向临床阶段。药明康德旗下专注于寡核苷酸、多肽及相关化学偶联药物的WuXi TIDES可以为复杂的寡核苷酸偶联药物提供一站式解决方案，支持从药物发现到开发再到商业化生产。全面的平台能力包括寡核苷酸、多肽和小分子，涵盖配体、毒素、连接子、脂质、聚乙二醇（PEG）等。凭借丰富的经验，WuXi TIDES能够提供灵活的解决方案，项目周转时间短，并可为任何规模的序列设计、合成以及分析提供全面的支持。在新型偶联药物不断发展的背景下，抗体偶联寡核苷酸（Antibody-Oligonucleotide Conjugates，AOCs）因兼具抗体的靶向递送能力与寡核苷酸的序列特异性调控优势，正成为突破组织特异性递送限制的重要技术方向。围绕这一复杂分子形式，药明康德DMPK团队建立了覆盖体外ADME研究、体内药代动力学（PK）评价及生物分析方法开发的系统化能力体系，可针对AOC由抗体、连接子与寡核苷酸三部分构成所带来的多维度代谢与分布挑战，设计匹配的研究策略。基于对AOC结构特征与ADME行为关系的深入理解，团队能够支持代谢物鉴定、暴露-效应关系分析以及PK研究方案优化，并为连接子类型选择、偶联位点设计及寡核苷酸修饰策略提供数据支持，从而在体内稳定性、靶向递送与细胞内释放之间实现平衡。依托跨平台检测技术与寡核苷酸DMPK研究经验的协同整合，该能力不仅能够解析AOC在ADME过程中的关键行为特征，也可贯穿发现、临床前及临床阶段，为客户在新型偶联药物开发中提供兼具科学深度与转化导向的一体化DMPK解决方案，加速复杂新分子从机制验证迈向临床转化。每一次递送技术更新，都可能让核酸药物触达新的患者 VERVE-102和z-rostudirsen代表的不只是两款候选药物的最新进展，而是核酸药物创新正在发生的变化。前者显示，肝脏靶向递送有望支持体内编辑工具进入慢病长期管理；后者显示，偶联递送正在推动PMO等寡核苷酸药物向肌肉和神经肌肉疾病场景深入。递送技术的每一次更新，都是寡核苷酸药物治疗边界的一次扩展。随着更多递送路径被验证，核酸药物也有望从相对成熟的肝脏疾病领域，走向肌肉、神经系统和更多复杂疾病场景。参考资料： [1] A single dose of Lilly's PCSK9 base editor, VERVE-102, reduced PCSK9 by up to 88% and LDL-C by up to 62%, with durable effects supporting its potential as a one-time treatment for hypercholesterolemia. Retrieved May 26, 2026, from https://investor.lilly.com/news-releases/news-release-details/single-dose-lillys-pcsk9-base-editor-verve-102-reduced-pcsk9-88 [2] Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD). Retrieved May 26, 2026, from https://www.globenewswire.com/news-release/2026/05/26/3300969/0/en/dyne-therapeutics-announces-submission-of-biologics-license-application-bla-to-u-s-fda-for-z-rostudirsen-in-exon-51-duchenne-muscular-dystrophy-dmd.html 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2026-05-26

·BioSpace

Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)

- Submission for Accelerated Approval based on dystrophin as a surrogate endpoint - - In the registrational expansion cohort of the DELIVER trial, treatment with z-rostudirsen resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile 1 - - Proposed dosing regimen of 20 mg/kg administered intravenously once every 4 weeks (Q4W) - WALTHAM, Mass., May 26, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc . (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) 20 mg/kg Q4W for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. “This is a significant milestone for both our company and the DMD community,” said John Cox, president and chief executive officer of Dyne. “Despite the availability of approved therapies, there remains a significant unmet need in DMD for treatments with compelling efficacy, a favorable safety pro improved dosing convenience. Z-rostudirsen was developed with the goal of delivering functional improvement with lower treatment burden for those living with this progressive disease. We look forward to continued engagement with the FDA to help facilitate a timely review, as we work toward bringing this potential best-in-class therapy to those in need as quickly as possible.” Dyne has requested Priority Review for the BLA, which, if granted, would shorten the review process from 10 months to 6 months following the FDA’s 60-day filing review period. Dyne continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming the FDA grants Priority Review and approval is received on the anticipated timeline. In addition to z-rostudirsen, Dyne is advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the potential treatment of DMD amenable to skipping of exons 53, 45, 44, and 55, respectively. About Zeleciment Rostudirsen (z-rostudirsen, also known as DYNE-251) Z-rostudirsen is an investigational therapeutic for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. The registrational expansion cohort of the global Phase 1/2 DELIVER clinical trial of z-rostudirsen met its primary endpoint. Data from the DELIVER trial served as the basis for a Biologics License Application (BLA) for potential U.S. Accelerated Approval. Z-rostudirsen continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial. Z-rostudirsen consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). It is designed to enable the production of near-full length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement. Z-rostudirsen has received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the FDA, European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan for the treatment of individuals with DMD amenable to exon 51 skipping. In addition to z-rostudirsen, Dyne is building a DMD franchise and has preclinical programs targeting other exons, including DYNE-253, DYNE-245, DYNE-244 and DYNE-255. About Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy (DMD) is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. These mutations result in a complete or near-complete absence of dystrophin, a protein critical for maintaining muscle structure and function. DMD is the most common form of childhood-onset muscular dystrophy, affecting approximately 12,000 individuals in the U.S. and 16,000 in the EU. Symptoms typically emerge between ages 3 and 5, beginning with muscle weakness in the upper arms, thighs and pelvic region, and progressively impacting the lower limbs, forearms, neck and trunk. In addition to physical decline, individuals may experience cognitive impairment and neuropsychiatric challenges such as intellectual disabilities, learning difficulties and behavioral disorders. Despite existing therapies, there remains a significant unmet need for new treatment options that deliver functional improvement. About Dyne Therapeutics Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease. The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more at , and follow us on X , LinkedIn and Facebook . Forward-Looking Statements 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 clinical and therapeutic potential of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), the potential of z-rostudirsen to be a best-in-class therapy, expectations regarding the timing and outcome of interactions with regulatory authorities and the availability of the Accelerated Approval pathway for z-rostudirsen, the potential availability of Priority Review, and expectations regarding the timing of commercialization of z-rostudirsen, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” “will” 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. 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 clinical trials; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne's clinical trials and the regulatory approval process; whether Dyne’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-Q and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne’s views as of any date subsequent to the date of this press release. Z-rostudirsen safety data as of August 19, 2025. Contacts: Investors Mia Tobias ir@dyne-tx.com 781-317-0353 Media Stacy Nartker snartker@dyne-tx.com 781-317-1938

临床3期优先审批突破性疗法孤儿药快速通道

100 项与泽雷西孟特・罗司地生相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
杜氏型和贝克型肌营养不良症	临床3期	美国	Dyne Therapeutics, Inc.	2026-06-01
杜氏肌营养不良症	临床3期	美国	Dyne Therapeutics, Inc.	2026-06-01

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05524883 (DELIVER, AAN2025)	临床1/2期	杜氏肌营养不良症 mutations amenable to exon 51 skipping	54	DYNE-251	網糧淵醖顧鹹鹹獵構壓(膚憲鬱鹹壓觸壓齋鏇鬱) = 繭鬱淵憲襯獵鏇鏇構淵鹹積遞遞襯鑰鏇構鹹獵 (鹽襯獵衊選獵憲顧構醖 ) 更多	积极	2025-04-07
NCT05524883 (DELIVER, MDA2025)	临床1/2期	杜氏肌营养不良症	54	DYNE-251 10 mg/kg	鑰製製獵淵衊構艱獵築(淵簾餘鑰餘築構積襯壓) = 糧選蓋選獵餘簾淵壓鹽鏇膚壓鏇鏇選壓壓鹽膚 (蓋憲鹽製遞鏇鬱鬱衊製 ) 更多	积极	2025-03-16
				DYNE-251 20 mg/kg	鑰製製獵淵衊構艱獵築(淵簾餘鑰餘築構積襯壓) = 艱繭衊遞醖範淵製糧築鏇膚壓鏇鏇選壓壓鹽膚 (蓋憲鹽製遞鏇鬱鬱衊製 ) 更多
NCT05524883 (DELIVER, Company_Website \| PipelineReview) 人工标引	临床1/2期	杜氏肌营养不良症 exon 51 skipping	54	DYNE-251	蓋獵鹽鹽獵衊鹽鑰繭夢(簾醖廠夢範鑰憲窪餘遞) = 鬱廠艱鹹糧餘膚構構窪齋壓醖壓鑰鹹製願製製 (醖鑰膚願餘鬱憲鹹鑰觸 )	积极	2024-09-03
				Placebo	-
NCT05524883 (DELIVER, Company_Website) 人工标引	临床1/2期	杜氏肌营养不良症	48	DYNE-251 5 mg/kg	鹽鏇願窪簾襯鬱鹽獵鹽(遞觸鹹糧淵襯糧獵淵壓) = 2 (4%) 觸願獵廠衊襯憲觸鑰願 (艱壓餘構醖壓憲鑰願範 ) 更多	积极	2024-05-20
				DYNE-251 10 mg/kg
NCT05524883 (DELIVER, Corporate Publications) 人工标引	临床1/2期	杜氏肌营养不良症	6	DYNE-251	壓鬱襯願遞選衊淵積顧(築鹹鑰憲窪膚壓簾鏇衊) = 選夢廠齋積築壓憲選築願艱餘鬱選壓醖廠醖鏇 (鑰廠醖顧餘蓋顧築蓋製 ) 更多	积极	2024-01-03
				Placebo	繭鹹糧襯鹹壓壓夢築廠(醖壓觸襯鹽願獵鹹壓鏇) = 廠顧簾願鑰顧積製構獵獵鬱鹹獵蓋繭遞遞壓憲 (願醖觸鹽窪餘鑰簾餘選 ) 更多