CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that screening is underway in Study SRP-9003-301. Also known as EMERGENE, Study 9003-301 is a Phase 3, multi-national, open-label study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy.

马萨诸塞州剑桥市（商业新闻短讯）--罕见疾病精密遗传医学的领导者Sarepta Therapeutics，Inc.（纳斯达克：SRPT）今天宣布，SRP-9003-301研究正在进行筛查。研究9003-301也称为EMERGENE，是一项针对SRP-9003（bidridistrogene xeboparvovec）的3期多国开放标签研究，用于治疗2E型肢带型肌营养不良症（LGMD2E/R4）或β-糖皮质病。

EMERGENE will enroll 15 participants (ambulatory and non-ambulatory), aged 4 and older, and uses commercially representative process SRP-9003 material..

EMERGENE将招募15名4岁及以上的参与者（非卧床和非卧床），并使用具有商业代表性的流程SRP-9003材料。。

“We are pleased to share our continued progress in advancing SRP-9003, our investigational gene therapy candidate for LGMD2E, a rare form of LGMD with no treatments beyond symptom management. Early results from the SRP-9003 clinical development program demonstrated significant protein expression at both 12-weeks and two years after treatment as well as functional benefits including slowing progression of this disease, improving mobility, and enhancing the quality of life for individuals living with LGMD2E,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics.

“我们很高兴分享我们在推进SRP-9003方面的持续进展，SRP-9003是LGMD2E的研究性基因治疗候选者，LGMD2E是一种罕见的LGMD形式，除了症状管理之外没有其他治疗方法。SRP-9003临床开发计划的早期结果表明，治疗后12周和两年蛋白质表达均显着，并且具有功能益处，包括减缓进展Sarepta Therapeutics执行副总裁、首席科学官兼研发负责人Louise Rodino Klapac博士说：“LGMD2E患者可以缓解这种疾病，改善活动能力，提高生活质量。”。

“In addition to its importance for the LGMD2E community, EMERGENE will inform the clinical development of other programs for LGMD in Sarepta’s pipeline while serving as a pathfinder for viable regulatory pathways to support the development of gene therapies to treat ultra rare diseases.”.

“除了对LGMD2E社区的重要性外，EMERGENE还将在Sarepta的管道中为LGMD的其他项目的临床开发提供信息，同时作为可行监管途径的开拓者，以支持开发治疗超罕见疾病的基因疗法。”。

A webinar for the limb-girdle community is planned, details will be shared at a future date.

计划为肢带社区举办一次网络研讨会，详细信息将在未来的某个日期共享。

About SRP-9003 (bidridistrogene xeboparvovec)

关于SRP-9003（双丙炔诺酮xeboparvovec）

SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat neuromuscular diseases. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications..

SRP-9003（bidridistrogene xeboparvovec）是一种研究性基因疗法，使用AAVrh74载体，该载体被设计为全身和稳健地递送至骨骼肌，diaphragm肌和心肌，使其成为治疗神经肌肉疾病的理想候选者。SRP-9003旨在传递全长β-糖聚糖转基因，并使用MHCK7启动子，该启动子因其在心脏中稳健表达的能力而被选择，这对于2E型肢带型肌营养不良症（LGMD2E）患者至关重要，也称为β-糖聚糖病和LGMDR4，其中许多人死于肺部或心脏并发症。。

About Study SRP-9003-301 (EMERGENE)

关于研究SRP-9003-301（紧急）

EMERGENE, Study 9003-301 is a Phase 3, multinational, open-label study of SRP-9003 for the treatment of LGMD2E in 15 ambulatory and non-ambulatory participants, ages 4 and older. The EMERGENE design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing.

紧急情况下，研究9003-301是SRP-9003的第三阶段多国开放标签研究，用于治疗15名4岁及以上的非卧床和非卧床参与者的LGMD2E。紧急设计包含了为期六个月的自然历史导入。主要终点是给药后60天β-糖聚糖的表达。

Other endpoints include functional measures through month 60 and safety..

其他终点包括第60个月的功能措施和安全性。。

About Limb-girdle Muscular Dystrophy

关于肢带型肌营养不良症

Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Sarepta’s six LGMD gene therapy programs in development include LGMD2E/R4, LGMD2D, LGMD2C, LGMD2B, LGMD2L and LGMD2A, which together represent more than 70 percent of known LGMD cases..

肢带型肌营养不良症是一种遗传性疾病，会导致进行性虚弱和消瘦，这种疾病始于臀部和肩膀周围的肌肉，然后发展为手臂和腿部的肌肉。Sarepta正在开发的六个LGMD基因治疗计划包括LGMD2E/R4，LGMD2D，LGMD2C，LGMD2B，LGMD2L和LGMD2A，它们合计占已知LGMD病例的70%以上。。

Patients with LGMD2E (beta-sarcoglycanopathy) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality.

LGMD2E（β-糖聚糖病）患者在10岁之前开始出现神经肌肉症状，例如跑步，跳跃和爬楼梯困难。该疾病是LGMD的常染色体隐性亚型，在青少年时期会发展为行走能力丧失，通常会导致早期死亡。

There are currently no disease modifying treatments for LGMD2E..

目前尚无针对LGMD2E的疾病缓解疗法。。

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 www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook..

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。欲了解更多信息，请访问www.sarepta.com或在Twitter、LinkedIn、Instagram和Facebook上关注我们。。

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

我们经常在我们网站www.sarepta.com的“为投资者”部分发布对投资者可能重要的信息。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

This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性声明”。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。诸如“相信”、“预期”、“计划”、“预期”、“将”、“可能”、“打算”、“准备”、“展望”、“潜力”、“可能”等词语以及类似的表达方式旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements relating to our future operations, technologies and scientific approaches, business plans, priorities, research and development programs; and the potential benefits of SRP-9003..

这些前瞻性声明包括但不限于与我们未来的运营、技术和科学方法、商业计划、优先事项、研究和发展计划有关的声明；以及SRP-9003的潜在好处。。

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: our data may not be sufficient for obtaining regulatory approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; we may not be able to execute on our business plans, including meeting our ex.

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：我们的数据可能不足以获得监管部门的批准；临床前和临床试验的成功，特别是如果基于少量患者样本，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或咨询委员会的建议不一致，或者可能不符合监管机构对候选产品安全性和有效性的批准要求；我们可能无法及时或根本无法遵守FDA的所有要求；FDA和其他监管机构的法规和监管决定可能对我们的业务产生的影响；我们的临床试验的开始和完成以及结果的宣布可能会因多种原因而延迟或阻止，其中包括监管机构拒绝批准继续进行我们的临床试验，或暂停临床试验，识别，招募，招募和保留患者参加临床试验的挑战，以及候选产品或进行临床试验所需的其他材料的供应数量或质量不足；我们用于评估特定安全性或有效性参数的不同方法，假设和应用可能会产生不同的统计结果，即使我们认为从候选产品的临床试验中收集的数据是积极的，这些数据可能不足以支持FDA或其他全球监管机构的批准；我们可能无法执行我们的商业计划，包括满足我们的前任。

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

上述任何风险都可能对公司的业务、经营业绩和Sarepta普通股的交易价格产生重大不利影响。有关Sarepta面临的风险和不确定性的详细描述，鼓励您查看Sarepta向SEC提交的文件。我们提醒投资者不要过分依赖本新闻稿中的前瞻性声明。

Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law..

除法律要求外，Sarepta不承担根据本协议日期后的事件或情况公开更新其前瞻性声明的任何义务。。