WALTHAM, Mass., May 20, 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 positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

马萨诸塞州沃尔瑟姆，2024年5月20日（环球通讯社）--Dyne Therapeutics，Inc.（Nasdaq: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改变生活的疗法，今天宣布了其正在进行的1/2期ACHERE试验Dyne-101在1型强直性肌营养不良（DM1）患者中的临床数据，以及正在进行的1/2期DELIVER试验Dyne-251在Duchenne肌营养不良（DMD）患者中的临床数据，这些患者可以跳过第51外显子。

New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles..

两项试验的新数据表明，对关键疾病生物标志物产生了令人信服的影响，并改善了多个功能终点和有利的安全性。。

“We are excited to report new clinical data from both our ACHIEVE and DELIVER trials demonstrating meaningful impact on key biomarkers and functional improvement in multiple clinical endpoints that matter to patients. We believe these data reflect the best-in-class potential for these product candidates and reinforce the opportunity to transform the treatment of DM1 and DMD as well as the potential of the FORCE platform to address other rare muscle diseases,” said John Cox, Dyne’s president and chief executive officer..

达因总裁兼首席执行官约翰·考克斯（JohnCox）表示：“我们很高兴报告来自我们的ACHIVE和DELIVER试验的新临床数据，这些数据证明了对关键生物标志物的有意义的影响以及对患者重要的多个临床终点的功能改善。我们相信这些数据反映了这些候选产品的最佳潜力，并加强了改变DM1和DMD治疗的机会，以及FORCE平台解决其他罕见肌肉疾病的潜力。”。。

“We believe the breadth and depth of these data are truly differentiating. Our robust preclinical work is translating into clinical benefit along with favorable safety profiles for both DYNE-101 and DYNE-251. In ACHIEVE, treatment with DYNE-101 demonstrated consistent, dose-dependent splicing correction, which led to an improvement in muscle strength, function, and patient reported outcomes.

“我们相信这些数据的广度和深度是真正不同的。我们强大的临床前工作正在转化为临床益处，以及DYNE-101和DYNE-251的良好安全性。在ACHIEVE中，用DYNE-101治疗表现出一致的剂量依赖性剪接校正，从而改善了肌肉力量，功能和患者报告的结果。

In DELIVER, treatment with DYNE-251 resulted in dystrophin expression that exceeded levels that have been reported for the standard of care for DMD as well as trends in functional improvement earlier than expected,” said Wildon Farwell, M.D., MPH, Dyne’s chief medical officer. “The DM1 and Duchenne communities have waited too long for new and better therapeutic options.

在DELIVER中，DYNE-251治疗导致肌营养不良蛋白的表达超过了DMD标准治疗报告的水平，并且功能改善的趋势早于预期，”DYNE首席医疗官Wildon Farwell医学博士说。“DM1和Duchenne社区等待新的更好的治疗选择已经太久了。

Building on the strength of these encouraging data and recent regulatory interactions, we look forward to continuing to engage with global regulatory authorities throughout this year to pursue expedited approval pathways and address the urgent unmet medical needs in these communities. We are thankful to the participants and clinicians in these trials and the communities for their continued partnership.”.

在这些令人鼓舞的数据和最近的监管互动的基础上，我们期待着在今年全年继续与全球监管机构合作，寻求快速的批准途径，并解决这些社区迫切未满足的医疗需求。我们感谢这些试验的参与者和临床医生以及社区的持续合作。”。

Phase 1/2 ACHIEVE Trial of DYNE-101 in DM1

DYNE-101在DM1中的1/2期实现试验

Efficacy Data

功效数据

Dyne reported efficacy data from 40 adult DM1 patients enrolled in the randomized, placebo-controlled multiple ascending dose (MAD) portion of the DYNE-101 ACHIEVE trial, including 12-month data from the 1.8 mg/kg Q4W (approximate ASO dose) cohort (n=16), 6-month data from the 3.4 mg/kg Q4W cohort (n=16), and 3-month data from the 5.4 mg/kg Q8W cohort (n=8)..

Dyne报告了40名成年DM1患者的疗效数据，这些患者参加了Dyne-101 ACHERE试验的随机，安慰剂对照的多次递增剂量（MAD）部分，包括1.8 mg/kg Q4W（近似ASO剂量）队列（n=16）的12个月数据，3.4 mg/kg Q4W队列（n=16）的6个月数据，以及5.4 mg/kg Q8W队列（n=8）的3个月数据。。

In the ACHIEVE trial, DYNE-101 demonstrated robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in multiple functional endpoints and patient reported outcomes.

在ACHIVE试验中，DYNE-101表现出强大的肌肉输送和剂量依赖性，一致的剪接校正，同时还显示出多个功能终点和患者报告结果的改善。

Key findings from ACHIEVE include:

ACHIEVE的主要发现包括：

Splicing

拼接

DYNE-101 continued to show dose dependent splicing correction as seen in earlier cohorts. Patients in the 5.4 mg/kg Q8W cohort had a 27% mean splicing correction from baseline across a broad, 22-gene panel at 3 months, with all participants demonstrating splicing correction.

如早期队列所示，DYNE-101继续显示剂量依赖性剪接校正。5.4 mg/kg Q8W队列中的患者在3个月时，在广泛的22个基因组中，从基线平均剪接校正率为27%，所有参与者均表现出剪接校正。

Function

函数

Myotonia (vHOT): DYNE-101 demonstrated an improvement in myotonia as measured by video hand opening time (vHOT) in all reported cohorts. The 1.8 mg/kg Q4W group had a mean 3.1 second benefit in myotonia at 3 months that increased to 4.4 seconds at 12 months. In addition, the 5.4 mg/kg Q8W cohort had a mean 4.5 second improvement in myotonia at 3 months..

肌强直（vHOT）：在所有报告的队列中，通过视频开门时间（vHOT）测量，DYNE-101表现出肌强直的改善。1.8 mg/kg Q4W组在3个月时肌强直平均获益3.1秒，在12个月时增加到4.4秒。此外，5.4 mg/kg Q8W队列在3个月时肌强直平均改善了4.5秒。。

Strength and Timed Assessments: DYNE-101 demonstrated an improvement in muscle strength as measured by Quantitative Myometry Testing (QMT), a test of muscle strength and fatigue, and early and sustained potential benefit in 10-Meter Walk/Run Test and 5 Times Sit to Stand Test.

力量和定时评估：DYNE-101通过定量肌力测试（QMT），肌肉力量和疲劳测试以及10米步行/跑步测试和5次坐立测试的早期和持续潜在益处，证明了肌肉力量的改善。

Patient Reported Outcomes (PROs)

患者报告的结果（PRO）

Myotonic Dystrophy Health Index (MDHI): DYNE-101 demonstrated an overall improvement in the MDHI and benefit in all 17 subscales, including those that assess peripheral muscles, central nervous system, and gastrointestinal measures. These represent some of the most burdensome manifestations of DM1 and daily quality of life issues for patients and their families..

强直性肌营养不良健康指数（MDHI）：DYNE-101显示MDHI总体改善，并在所有17个分量表中受益，包括评估外周肌肉，中枢神经系统和胃肠道指标的分量表。这些代表了DM1的一些最繁重的表现，以及患者及其家人的日常生活质量问题。。

Myotonic Dystrophy Type 1 Activity and Participation Scale (DM1-ACTIVc): Treatment with DYNE-101 resulted in an improvement in the assessment of various activities of daily living, as measured by DM1-ACTIVc.

强直性肌营养不良1型活动和参与量表（DM1 ACTIVc）：用DYNE-101治疗可改善DM1 ACTIVc对各种日常生活活动的评估。

Safety and Tolerability Data

安全性和耐受性数据

Dyne also reported safety and tolerability data from 56 patients enrolled through the 6.8 mg/kg Q8W cohort of the MAD portion of the ACHIEVE trial. DYNE-101 demonstrated a favorable safety profile.2 The majority of treatment emergent adverse events were mild or moderate and no related serious treatment emergent adverse events have been identified..

达因还报告了56名患者的安全性和耐受性数据，这些患者是通过ACHIVE试验MAD部分的6.8 mg/kg Q8W队列登记的。DYNE-101表现出良好的安全性。2大多数治疗紧急不良事件为轻度或中度，未发现相关的严重治疗紧急不良事件。。

Enrollment is complete through the 6.8 mg/kg Q8W cohort. Approximately 500 doses have been administered to date, representing over 40-patient years of follow-up and supporting dose escalation up to 10.2 mg/kg.

通过6.8 mg/kg Q8W队列完成登记。迄今为止，已经服用了大约500剂，代表了40多年的随访，并支持剂量增加至10.2 mg/kg。

Phase 1/2 DELIVER Trial of DYNE-251 in DMD

DYNE-251在DMD中的1/2期交付试验

Efficacy Data

功效数据

Dyne reported efficacy data from 8 male patients with DMD amenable to exon 51 skipping enrolled in the 10 mg/kg (approximate PMO dose) cohort of the randomized, placebo-controlled MAD portion of the DYNE-251 DELIVER trial. Patients were randomized to receive either DYNE-251 (n=6) or placebo (n=2) once every four weeks for 6 months..

Dyne报告了8名男性DMD患者的疗效数据，这些患者可以跳过外显子51，这些患者参加了Dyne-251 DELIVER试验的随机安慰剂对照MAD部分的10 mg/kg（近似PMO剂量）队列。患者被随机分配接受DYNE-251（n=6）或安慰剂（n=2），每四周一次，持续6个月。。

10 mg/kg of DYNE-251 Q4W demonstrated dose-dependent exon skipping and dystrophin expression. DYNE-251 reached levels of dystrophin expression that exceeded levels reported in a clinical trial for the current weekly standard of care for DMD exon 51, eteplirsen, at 6 months1 with a 12-fold lower PMO dose.

10 mg/kg的DYNE-251 Q4W表现出剂量依赖性外显子跳跃和肌营养不良蛋白表达。DYNE-251在6个月时达到了肌营养不良蛋白表达水平，超过了目前DMD外显子51 eteplirsen每周护理标准的临床试验报告的水平1，PMO剂量降低了12倍。

DYNE-251 also demonstrated encouraging trends in multiple functional endpoints..

DYNE-251在多个功能终点方面也表现出令人鼓舞的趋势。。

Key findings from DELIVER include:

DELIVER的主要发现包括：

Dystrophin expression measured by Western blot

Western blot检测肌营养不良蛋白的表达

Patients treated with 10 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin level of 3.22% of normal and a 2.97% change (unadjusted for muscle content) from baseline at 6 months. Eteplirsen reached a mean absolute unadjusted dystrophin level of 0.30% of normal and a 0.06% change from baseline at 6 months.1 When adjusting for muscle content, the DYNE-251 treated group reached 7.64% mean absolute dystrophin, which is greater than levels reported by peptide conjugate PMOs in clinical development.3.

接受10 mg/kg DYNE-251 Q4W治疗的患者在6个月时的平均绝对肌营养不良蛋白水平为正常水平的3.22%，与基线相比变化了2.97%（未调整肌肉含量）。Eteplirsen在6个月时达到平均绝对未调整的肌营养不良蛋白水平，为正常水平的0.30%，与基线相比变化为0.06%[1]。调整肌肉含量时，DYNE-251治疗组的平均绝对肌营养不良蛋白达到7.64%，高于肽缀合物PMOs在临床开发中报道的水平。

Function

函数

Trends in improvement were observed in multiple functional endpoints in the 10 mg/kg DYNE-251 Q4W group at 6 months, including North Star Ambulatory Assessment (NSAA), Stride Velocity 95th Centile (SV95C), 10-Meter Walk/Run Time, and Time to Rise from Floor.

在6个月时，在10 mg/kg DYNE-251 Q4W组的多个功能终点中观察到改善趋势，包括北极星动态评估（NSAA），步速第95百分位（SV95C），10米步行/跑步时间和从地板上升的时间。

Safety and Tolerability Data

安全性和耐受性数据

Dyne also reported safety and tolerability data from 48 patients enrolled through the 40 mg/kg Q8W cohort of the MAD portion of the DELIVER trial. DYNE-251 demonstrated a favorable safety profile.4 The majority of treatment emergent adverse events were mild or moderate and no related serious treatment emergent adverse events have been identified..

Dyne还报告了48名患者的安全性和耐受性数据，这些患者是通过DELIVER试验MAD部分的40 mg/kg Q8W队列登记的。DYNE-251表现出良好的安全性。4大多数治疗紧急不良事件为轻度或中度，未发现相关的严重治疗紧急不良事件。。

Enrollment is complete through the 40 mg/kg cohort. Approximately 480 doses have been administered to date, representing over 35 patient-years of follow-up, supporting dose escalation up to 40 mg/kg.

通过40 mg/kg队列完成登记。迄今为止，已经服用了大约480剂，代表了超过35个患者年的随访，支持剂量增加至40 mg/kg。

Key Milestones

关键里程碑

ACHIEVE and DELIVER Trials

实现并交付试用

Dyne plans to continue to engage with global regulators this year on ACHIEVE and DELIVER, and anticipates providing an update on the path to registration for both DYNE-101 and DYNE-251 by the end of 2024. Both trials are designed to be registrational, and the company is pursuing expedited approval pathways for both programs..

Dyne计划今年继续与全球监管机构接洽ACHIEVE and DELIVER，并预计在2024年底前提供Dyne-101和Dyne-251注册路径的最新信息。这两项试验都是为了注册而设计的，该公司正在为这两个项目寻求快速的批准途径。。

In DM1, based on recent dialogue with the Center for Drug Evaluation and Research (CDER) division of the U.S. Food and Drug Administration (FDA), Dyne continues to pursue an accelerated approval pathway for DYNE-101, including leveraging splicing as a potential surrogate biomarker.

在DM1中，根据最近与美国食品和药物管理局（FDA）药物评估和研究中心（CDER）部门的对话，Dyne继续寻求Dyne-101的加速批准途径，包括利用剪接作为潜在的替代生物标志物。

In DMD, Dyne has confirmed that the FDA precedent for using dystrophin as a surrogate biomarker for accelerated approval remains available.

在DMD中，达恩证实，FDA使用肌营养不良蛋白作为加速批准的替代生物标志物的先例仍然可用。

FORCE™ Platform and Pipeline

FORCE™平台和管道

With ACHIEVE and DELIVER data continuing to reinforce the promise of the FORCE platform, Dyne expects to provide updates on programs, including facioscapulohumeral muscular dystrophy (FSHD) and other pipeline opportunities during 2024.

随着实现和交付数据不断增强FORCE平台的前景，Dyne预计将在2024年提供有关计划的更新，包括面肩肱型肌营养不良症（FSHD）和其他管道机会。

Virtual Investor Event

虚拟投资者活动

Dyne will host a live video webcast event to discuss these ACHIEVE and DELIVER data today, May 20, 2024, at 8:00 a.m. ET. The live webcast will be available on the Events & Presentations page of the Investors & Media section of Dyne’s website and a replay will be accessible for 90 days following the presentation.

Dyne将于2024年5月20日（美国东部时间今天上午8:00）主持一场现场视频网络直播活动，讨论这些实现并交付数据。现场网络直播将在Dyne网站投资者和媒体部分的活动和演示页面上进行，演示后90天内可以进行重播。

An accompanying slide presentation will also be available. To access the presentation, register for the live webcast and replay, please visit https://investors.dyne-tx.com/news-and-events/events-and-presentations..

还将提供随附的幻灯片演示。要访问演示文稿，请注册网络直播和重播，请访问https://investors.dyne-tx.com/news-and-events/events-and-presentations..

About ACHIEVE

关于实现

ACHIEVE is a Phase 1/2 global clinical trial evaluating DYNE-101, consisting of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is enrolling adult patients with myotonic dystrophy type 1 (DM1) who are 18 to 49 years of age.

ACHIVE是一项评估DYNE-101的1/2期全球临床试验，包括24周的多次递增剂量（MAD）随机安慰剂对照期，24周的开放标签延长和96周的长期延长。该试验旨在注册，正在招募18至49岁的1型强直性肌营养不良（DM1）成年患者。

The primary endpoints are safety and tolerability, with secondary endpoints of pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function. For more information on the ACHIEVE trial, visit https://www.clinicaltrials.gov/ (NCT05481879)..

主要终点是安全性和耐受性，其次是药代动力学和药效学终点，包括剪接基线的变化，以及肌肉力量和功能的测量。有关实现试验的更多信息，请访问https://www.clinicaltrials.gov/（NCT05481879）。。

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 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.

它旨在实现靶向肌肉组织递送，目的是减少细胞核中有毒的DMPK 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已被美国授予孤儿药称号。

Food and Drug Administration and the European Medicines Agency for the treatment of DM1..

食品和药物管理局和欧洲药品管理局治疗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 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.

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。它是一种单基因常染色体显性遗传疾病，由DMPK基因区域的异常三核苷酸扩增引起。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。

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. 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..

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。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 the DELIVER Trial

关于交付试用

DELIVER is a Phase 1/2 global clinical trial evaluating DYNE-251, consisting of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is enrolling ambulant and non-ambulant males with Duchenne muscular dystrophy (DMD) who are ages 4 to 16 and have mutations amenable to exon 51 skipping.

DELIVER是一项评估DYNE-251的1/2期全球临床试验，包括24周的多次递增剂量（MAD）随机安慰剂对照期，24周的开放标签延长和96周的长期延长。该试验旨在注册，正在招募4至16岁的杜氏肌营养不良症（DMD）的非卧床和非卧床男性，他们的突变适合外显子51跳跃。

The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping and pharmacokinetics. For more information on the DELIVER trial, visit https://www.clinicaltrials.gov/ (NCT05524883)..

主要终点是通过蛋白质印迹测量的肌营养不良蛋白水平的安全性，耐受性和基线变化。次要终点包括肌肉功能，外显子跳跃和药代动力学的测量。有关DELIVER试用的更多信息，请访问https://www.clinicaltrials.gov/（NCT05524883）。。

About DYNE-251

关于DYNE-251

DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle.

DYNE-251是一种研究性治疗药物，正在1/2期全球交付临床试验中进行评估，用于适合外显子51跳跃的DMD患者。DYNE-251由与片段抗体（Fab）偶联的磷酸二酰胺吗啉代低聚物（PMO）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 and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S.

它旨在实现靶向肌肉组织递送并促进细胞核中外显子跳跃，从而使肌肉细胞产生截短的功能性肌营养不良蛋白，目的是阻止或逆转疾病进展。DYNE-251已被美国授予快速通道、孤儿药和罕见儿科疾病称号。

Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping..

美国食品和药物管理局（FDA）用于治疗可跳过外显子51的DMD突变。。

In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.

除DYNE-251外，DYNE正在建立全球DMD特许经营权，并针对其他外显子（包括53、45和44）进行临床前计划。

About Duchenne Muscular Dystrophy (DMD)

关于杜兴氏肌营养不良症（DMD）

DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S.

DMD是一种罕见疾病，由编码肌营养不良蛋白的基因突变引起，肌营养不良蛋白是一种对肌肉细胞正常功能至关重要的蛋白质。这些突变（其中大多数是缺失）导致肌营养不良蛋白缺乏和肌肉功能逐渐丧失。DMD主要发生在男性中，在美国估计有12000至15000人受到影响。

and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens.

欧洲有25000人。力量和功能的丧失通常首先出现在学龄前男孩身上，并随着年龄的增长而恶化。随着疾病的进展，骨骼肌和心肌损伤的严重程度通常会导致患者在青少年早期完全丧失行走能力，包括心脏和呼吸系统症状恶化以及青少年后期上身功能丧失。

There is no cure for DMD and currently approved therapies provide limited benefit..

DMD没有治愈方法，目前批准的疗法提供的益处有限。。

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。。