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)--Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy..

)--罕见疾病精密遗传医学的领导者Sarepta Therapeutics，Inc.（纳斯达克股票代码：SRPT）今天宣布，EMBARK（研究SRP-9001-301）第2部分的结果为阳性，EMBARK是一项全球性，随机，双盲，安慰剂对照的ELEVIDYS（delandistrogene moxeparvovec rokl）3期临床研究，是唯一批准用于杜兴氏肌营养不良症患者的基因治疗。。

Crossover-treated patients, those who received a placebo in Part 1 and crossed over at 52 weeks and were treated with ELEVIDYS in Part 2, improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment (P<0.0001), during which time the study remained blinded..

交叉治疗的患者，即在第1部分接受安慰剂治疗并在第52周交叉治疗并在第2部分接受ELEVIDYS治疗的患者，在治疗52周后，与北极星动态评估（NSAA）的匹配外部对照组相比，比基线改善了2.34分（P<0.0001），在此期间，研究仍然是盲目的。。

Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years), crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) function tests compared with a pre-specified, propensity-weighted external control group* (EC)..

尽管交叉治疗的患者比第1部分治疗的患者（平均年龄5.98岁）大一岁（平均年龄7.18岁），但与预先指定的倾向加权外部对照组*（EC）相比，交叉治疗的患者对NSAA，上升时间（TTR）和10米步行/跑步（10MWR）功能测试显示出临床意义和统计学显着的功能益处。。

Crossover-Treated Patients (n=59) vs. EC

交叉治疗患者（n=59）与EC

Functional Outcomes

功能结果

LSM

LSM

P-Value

P值

NSAA

NSAA

+2.34 points

+2.34分

P<0.0001

P<0.0001

TTR

TTR

-2.70 seconds (improvement)

-2.70秒（改进）

P<0.0001

P<0.0001

10MWR

10MWR

-1.07 seconds (improvement)

-1.07秒（改进）

P=0.0001

P=0.0001

Two-Year Results

两年业绩

In patients treated in Part 1, biopsies taken 64 weeks after dosing showed consistent and sustained expression of ELEVIDYS micro-dystrophin compared to week 12 biopsies, as measured by western blot, and provide biological support for observed functional outcomes.

在第1部分治疗的患者中，给药后64周的活组织检查显示，与第12周的活组织检查相比，ELEVIDYS微肌营养不良蛋白的表达持续一致，并为观察到的功能结果提供了生物学支持。

At two years, patients treated in Part 1 of EMBARK showed clinically meaningful and statistically significant functional benefit in NSAA, TTR and 10MWR compared with EC. Furthermore, the least square means (LSM) differences seen between the patients treated in Part 1 and the EC group increase from year one to year two for all three functional outcomes.

两年时，与EC相比，EMBARK第1部分治疗的患者在NSAA，TTR和10MWR方面显示出临床意义和统计学显着的功能益处。此外，第1部分治疗的患者与EC组之间的最小二乘平均值（LSM）差异从第一年增加到第二年，所有三种功能结果。

This indicates that the trajectory of disease in patients treated with ELEVIDYS is continuing to diverge from the natural history of DMD..

这表明用ELEVIDYS治疗的患者的疾病轨迹继续偏离DMD的自然史。。

Part 1, Year 2 (n=63) ELEVIDYS-Treated vs. EC

第1部分，第2年（n=63）ELEVIDYS治疗与EC

Functional Outcomes

功能结果

LSM

LSM

P-Value

P值

NSAA

NSAA

+2.88 points

+

P=0.0001

P=0.0001

TTR

TTR

-2.06 seconds (improvement)

-2.06秒（改进）

P=0.0033

P=0.0033

10MWR

10兆瓦

-1.36 seconds (improvement)

-1.36秒（改进）

P=0.0028

P=0.0028

Skeletal muscle MRI conducted on patients treated in Part 1 found minimal progression in underlying muscle pathology and remain highly consistent with the functional benefits shown.

对第1部分治疗的患者进行的骨骼肌MRI发现，潜在的肌肉病理学进展最小，并且与所显示的功能益处高度一致。

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We’re very encouraged to see the results from Part 2 of EMBARK as they further elucidate the impact ELEVIDYS has on disease progression in a blinded, controlled study. Skeletal muscle MRI demonstrates the importance of preserving muscle, and the functional outcome results show disease stabilization sustained through two years after treatment,” said Louise Rodino-Klapac, Ph.D., executive vice president, Head of R&D, Chief Scientific Officer.

我们很高兴看到EMBARK第2部分的结果，因为他们在一项盲法对照研究中进一步阐明了ELEVIDYS对疾病进展的影响。骨骼肌MRI证明了保留肌肉的重要性，功能结果显示治疗后两年内疾病稳定，”首席科学官、研发主管、执行副总裁路易丝·罗迪诺·克拉帕克博士说。

“.

“.

Over time, we continue to observe a statistically significant difference favoring ELEVIDYS compared to a well-matched external control on NSAA and timed tests. The consistency and totality of evidence supporting a long-term and clinically meaningful treatment benefit with ELEVIDYS continues to grow.

随着时间的推移，我们继续观察到与NSAA和定时测试上匹配良好的外部对照相比，有利于ELEVIDYS的统计学显着差异。支持ELEVIDYS长期和临床上有意义的治疗益处的证据的一致性和整体性继续增长。

We look forward to sharing more details with the clinical community in upcoming scientific forums.”.

我们期待在即将到来的科学论坛上与临床界分享更多细节。”。

No new safety signals were observed, reinforcing the consistent and manageable safety profile of ELEVIDYS to date. Detailed results from Part 2 of the EMBARK study will be shared at future medical meetings.

没有观察到新的安全信号，加强了迄今为止ELEVIDYS的一致性和可管理的安全性。EMBARK研究第2部分的详细结果将在未来的医学会议上分享。

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“

As a neuromuscular medicine specialist who has seen patients with Duchenne muscular dystrophy for over three decades, I’ve witnessed firsthand the positive impact of gene therapy on the trajectory of Duchenne,” said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, and an investigator in the EMBARK study.

作为一名神经肌肉医学专家，三十多年来一直在观察杜兴氏肌营养不良症患者，我亲眼目睹了基因治疗对杜兴氏轨迹的积极影响，”加州大学戴维斯分校物理医学与康复系教授兼主席、EMBARK研究的研究员克雷格·麦克唐纳（CraigMcDonald）医学博士说。

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“.

These longer-term results are even more striking when compared to external control given the progressive nature of the disease, and we’d expect to see this divergence grow over time. The efficacy of ELEVIDYS gives me great hope as we continue to follow these patients and see others treated in the clinical setting.”.

考虑到疾病的进展性质，与外部控制相比，这些长期结果更加引人注目，我们预计这种分歧会随着时间的推移而增长。ELEVIDYS的疗效给了我很大的希望，因为我们继续跟踪这些患者，并看到其他患者在临床环境中接受治疗。”。

As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing.

作为2019年签署的合作协议的一部分，萨雷塔正在与罗氏合作，改变杜兴社区的未来，使患有这种疾病的人能够维持和保护他们的肌肉功能。Sarepta负责ELEVIDYS在美国的监管批准和商业化以及制造。

Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world..

罗氏负责监管部门的批准，并将ELEVIDYS带给世界各地的患者。。

ELEVIDYS is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the U.S., United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. ELEVIDYS is also approved for the treatment of ambulatory patients aged four through seven years in Brazil and Israel..

在美国、阿拉伯联合酋长国（UAE）、卡塔尔、科威特、巴林和阿曼，ELEVIDYS适用于与四岁及以上的Duchenne同住的人，无论他们的门诊状况如何。ELEVIDYS还被批准用于治疗巴西和以色列4至7岁的门诊患者。。

*The pre-specified external control used data from five separate studies in Duchenne, comprising DMD controls from two randomized trials and three natural history cohorts who met predefined matching criteria. Comparison of treated and control patients was based on a pre-specified, propensity score weighting approach using age, steroid usage, baseline NSAA and timed function tests in order to balance key prognostic factors between the groups..

*预先指定的外部对照使用了来自Duchenne的五项独立研究的数据，包括来自两项随机试验的DMD对照和符合预定义匹配标准的三个自然史队列。治疗患者和对照患者的比较基于预先指定的倾向评分加权方法，使用年龄，类固醇使用，基线NSAA和定时功能测试来平衡组间的关键预后因素。。

Sarepta Investor Call Details

Sarepta投资者电话详情

At 8:30 a.m. ET on Jan. 27, 2025, Sarepta will host a conference call and webcast to discuss these results.

2025年1月27日美国东部时间上午8:30，萨雷塔将主持电话会议和网络广播，讨论这些结果。

The event will be webcast live under the investor relations section of Sarepta’s website at

该活动将在Sarepta网站的投资者关系部分进行在线直播

https://investorrelations.sarepta.com/events-presentations

https://investorrelations.sarepta.com/events-presentations

and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using

活动结束后，重播将在那里存档一年。通过电话参与的相关方需要使用

this online form

此在线表单

. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

。在注册拨入详细信息后，所有电话参与者将收到一封自动生成的电子邮件，其中包含拨入号码的链接以及用于通过电话访问活动的个人PIN号码。

About EMBARK, Study 9001-301

关于登机，研究9001-301

Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment.

研究SRP-9001-301，也称为EMBARK，是一项针对4至7岁杜兴氏肌营养不良症患者的ELEVIDYS的跨国，3期，随机，两部分交叉，安慰剂对照研究。主要终点是治疗后第52周NSAA总分与基线的变化。

Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study..

符合条件的参与者在研究的第一部分或第二部分接受了单剂量的ELEVIDYS。。

In Part 1, participants (n=125) were randomized according to age (≥4 to <8 years) or NSAA Total Score at screening (>16 to <29) and received either 1.33 x1014 vg/kg of ELEVIDYS or placebo with a follow-up period for 52 weeks. In Part 2, participants cross over - meaning, those who were previously treated with placebo in Part 1 receive ELEVIDYS and participants who were previously treated with ELEVIDYS receive placebo, with a follow-up period for 52 weeks.

在第1部分中，参与者（n=125）根据年龄（≥4至8岁）或筛查时的NSAA总分（>16至29）随机分组，并接受1.33×1014 vg/kg的ELEVIDYS或安慰剂，随访52周。。

All patients remained blinded through Part 1 and Part 2..

所有患者在第1部分和第2部分仍然不知情。。

Secondary outcome measures in EMBARK include the quantity of shortened dystrophin produced by ELEVIDYS at week 12 as measured by western blot in a subset of participants, timed function tests, stride velocity and validated patient reported outcome measures for mobility and upper limb function. One-year results from the Part 1 placebo-controlled period of the EMBARK study were published in .

EMBARK的次要结局指标包括第12周ELEVIDYS产生的肌营养不良蛋白缩短的数量，通过western blot在一部分参与者中进行测量，定时功能测试，步速以及经过验证的患者报告的活动性和上肢功能结局指标。EMBARK研究第一部分安慰剂对照期的一年结果发表在。

Nature Medicine

自然医学

in October 2024.

2024年10月。

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

关于ELEVIDYS（delndistrogen moxeparvovec-rokl）

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle..

。。

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

ELEVIDYS适用于治疗至少4岁的杜氏肌营养不良症（DMD）。

For patients who are ambulatory and have a confirmed mutation in the DMD gene

For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

对于非门诊且DMD基因突变已确认的患者。

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s)..

基于ELEVIDYS微肌营养不良蛋白（以下称为“微肌营养不良蛋白”）在骨骼肌中的表达，非门诊患者的DMD适应症在加速批准下获得批准。是否继续批准该适应症可能取决于验证性试验中临床益处的验证和描述。。

IMPORTANT SAFETY INFORMATION

重要安全信息

CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

禁忌症：对于DMD基因第8外显子和/或第9外显子缺失的患者，禁止使用ELEVIDYS。

WARNINGS AND PRECAUTIONS:

警告和注意事项：

Infusion-related Reactions:

输液相关反应：

Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment.

ELEVIDYS给药后数小时内或数小时内发生了输注相关反应，包括超敏反应和过敏反应。在给药期间和输注结束后至少3小时密切监测患者。如果出现输液相关反应的症状，请减慢或停止输液并给予适当的治疗。

Once symptoms resolve, the infusion may be restarted at a lower rate..

一旦症状消失，输液可能会以较低的速度重新开始。。

ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.

ELEVIDYS应该在可以立即治疗输液相关反应的环境中使用。

Discontinue infusion for anaphylaxis.

停止输注过敏反应。

Acute Serious Liver Injury:

急性严重肝损伤：

Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.

ELEVIDYS已观察到急性严重肝损伤，给药可能导致肝酶（如GGT，GLDH，ALT，AST）或总胆红素升高，通常在8周内出现。

Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

先前存在肝功能损害，慢性肝病或急性肝病（例如急性肝病毒感染）的患者可能有更高的急性严重肝损伤风险。推迟急性肝病患者的ELEVIDYS给药，直到解决或控制。

Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels)..

在ELEVIDYS给药之前，在ELEVIDYS输注后的前3个月内，每周进行肝酶测试并监测肝功能（临床检查，GGT和总胆红素）。如果临床指示，继续监测，直到结果不明显（正常临床检查，GGT和总胆红素水平恢复到接近基线水平）。。

Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

建议在ELEVIDYS输注前后对患者进行全身皮质类固醇治疗。根据需要调整皮质类固醇治疗方案。如果怀疑急性严重肝损伤，建议咨询专家。

Immune-mediated Myositis:

In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.

在临床试验中，在ELEVIDYS输注后约1个月，在DMD基因第8外显子和/或第9外显子缺失突变的患者中观察到免疫介导的肌炎。观察到严重肌肉无力的症状，包括吞咽困难，呼吸困难和声音不足。

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.

对于外显子1至17和/或外显子59至71中DMD基因突变的患者，ELEVIDYS治疗的数据有限。这些区域缺失的患者可能有发生严重免疫介导的肌炎反应的风险。

Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur..

如果患者出现任何无法解释的肌肉疼痛，压痛或无力增加，包括吞咽困难，呼吸困难或声音不足，建议患者立即联系医生，因为这些可能是肌炎的症状。如果出现这些症状，请根据患者的临床表现和病史考虑额外的免疫调节治疗（免疫抑制剂[例如钙调神经磷酸酶抑制剂]以及皮质类固醇）。。

Myocarditis:

心肌炎：

Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.

在临床试验中，ELEVIDYS输注后观察到急性严重心肌炎和肌钙蛋白-I升高。

If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath..

如果患者患有心肌炎，则先前存在左心室射血分数（LVEF）损伤的患者可能有更高的不良后果风险。在ELEVIDYS输注前和输注后第一个月每周监测肌钙蛋白-I，并在临床指示的情况下继续监测。如果存在心脏症状，例如胸痛或呼吸急促，可能需要更频繁的监测。。

Advise patients to contact a physician immediately if they experience cardiac symptoms.

。

Preexisting Immunity against AAVrh74:

先前存在的针对AAVrh74的免疫力：

In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.

在基于AAV载体的基因疗法中，先前存在的抗AAV抗体可能会在所需的治疗水平上阻碍转基因表达。用ELEVIDYS治疗后，所有患者均产生了抗AAVrh74抗体。

Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.

在ELEVIDYS给药之前，对抗AAVrh74总结合抗体的存在进行基线测试。

ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

对于抗AAVrh74总结合抗体滴度大于或等于1:400的患者，不建议使用ELEVIDYS。

Adverse Reactions:

不良反应：

The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

临床研究中报告的最常见不良反应（发生率≥5%）是呕吐，恶心，肝损伤，发热和血小板减少症。

Report negative side effects of prescription drugs to the FDA. Visit

向FDA报告处方药的负面副作用。访问

www.fda.gov/medwatch

政府/医疗观察

or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

。您也可以在1-888-Sarepta（1-888-727-3782）向Sarepta Therapeutics报告副作用。

For further information, please see the full

有关更多信息，请参阅完整

Prescribing Information

处方信息

.

.

About Sarepta Therapeutics

关于Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.

萨雷塔正在执行一项紧急任务：为罕见疾病设计精确的基因医学，这些疾病会破坏生命并缩短未来。我们在杜兴氏肌营养不良症（DMD）和肢带型肌营养不良症（LGMD）方面担任领导职务，目前我们有40多个项目处于不同的发展阶段。

Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit .

我们在基因治疗，RNA和基因编辑方面的多平台精密遗传医学引擎推动了我们庞大的管道。有关更多信息，请访问。

www.sarepta.com

www.sarepta.com

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Internet Posting of Information

网上发布信息

We routinely post information that may be important to investors in the 'For Investors' section of our website at

我们经常在我们网站的“为投资者”部分发布对投资者可能重要的信息

www.sarepta.com

www.sarepta.com

. We encourage investors and potential investors to consult our website regularly for important information about us.

。我们鼓励投资者和潜在投资者定期咨询我们的网站，以获取有关我们的重要信息。

Forward-Looking Statements

前瞻性声明

In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements.

为了让Sarepta的投资者了解其当前业绩和未来前景，本新闻稿包含前瞻性声明。本新闻稿中包含的任何不属于历史事实声明的声明都可能被视为前瞻性声明。

Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to ELEVIDYS; the potential benefits of our agreements with strategic partners; and expected milestones and plans, including sharing more details with the clinical community in upcoming scientific forums..

诸如“相信”，“预期”，“计划”，“预期”，“将”，“可能”，“打算”，“准备”，“期待”，“潜在”，“可能”等词语以及类似的表达方式旨在识别前瞻性陈述。这些前瞻性声明包括与ELEVIDYS有关的声明；我们与战略合作伙伴达成协议的潜在好处；以及预期的里程碑和计划，包括在即将到来的科学论坛上与临床社区分享更多细节。。

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: success in clinical trials does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review..

这些前瞻性陈述涉及风险和不确定性，其中许多超出了Sarepta的控制范围。由于此类风险和不确定性，实际结果可能与这些前瞻性声明所述或暗示的结果存在重大差异。已知的风险因素包括：临床试验的成功并不能确保以后的临床试验取得成功，未来研究的结果可能与过去的积极结果不一致；我们可能无法执行我们的业务计划，包括满足我们预期或计划的监管里程碑和时间表，由于各种原因的研究和临床开发计划，其中一些可能超出我们的控制范围，包括公司财务和其他资源的可能限制，可能无法及时预期或解决的制造限制，以及监管，法院或机构的决定，例如美国专利和商标局关于涵盖我们产品候选专利的决定；以及我们最近向美国证券交易委员会（SEC）提交的10-Q表季度报告中“风险因素”标题下确定的风险，以及鼓励您审查的该公司提交的其他SEC文件。。

About Sarepta Therapeutics

关于Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.

萨雷塔正在执行一项紧急任务：为罕见疾病设计精确的基因医学，这些疾病会破坏生命并缩短未来。我们在杜兴氏肌营养不良症（DMD）和肢带型肌营养不良症（LGMD）方面担任领导职务，目前我们有40多个项目处于不同的发展阶段。

Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit .

我们在基因治疗，RNA和基因编辑方面的多平台精密遗传医学引擎推动了我们庞大的管道。有关更多信息，请访问。

www.sarepta.com

www.sarepta.com

or follow us on

或者继续关注我们

LinkedIn

LinkedIn

,

,

X

十

,

,

Instagram

Instagram

and

和

Facebook

脸书

.

.

Internet Posting of Information

网上发布信息

We routinely post information that may be important to investors in the 'For Investors' section of our website at

我们经常在我们网站的“为投资者”部分发布对投资者可能重要的信息

www.sarepta.com

www.sarepta.com

. We encourage investors and potential investors to consult our website regularly for important information about us.

。我们鼓励投资者和潜在投资者定期咨询我们的网站，以获取有关我们的重要信息。