REGENXBIO宣布发表临床前结果，展示RGX-202研究性基因疗法中新型微抗肌萎缩蛋白构建体在杜氏肌营养不良症治疗中的功能益处-动脉网

REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy

CISION 等信源发布 2025-07-10 23:54

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Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin

包含CT结构域的构建体在缺乏dystrophin的小鼠中表现出更高水平的微dystrophin蛋白、增强的肌肉力量以及更好的抗损伤能力。

REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain

REGENXBIO的下一代研究性基因疗法RGX-202是唯一包含CT结构域的微营养不良蛋白构建体。

Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE

研究结果支持了在I/II期AFFINITY DUCHENNE试验中观察到的积极功能性数据

trial of RGX-202

RGX-202试验

ROCKVILLE, Md.

罗克维尔，马里兰州

，

July 10, 2025

2025年7月10日

/PRNewswire/ -- REGENXBIO Inc. (Nasdaq:

/PRNewswire/ -- REGENXBIO Inc.（纳斯达克：

RGNX

) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) domain to a microdystrophin construct without the CT domain. The results, which were published in peer-reviewed journal

）今天宣布了发表的临床前结果，比较了包含C端（CT）结构域的微营养不良蛋白基因治疗构建体与不含CT结构域的微营养不良蛋白构建体。这些结果发表在经同行评审的期刊上。

Molecular Therapy Methods and Clinical Development

分子治疗学方法与临床发展

, showed that the microdystrophin with the CT domain improved functional benefit compared to the microdystrophin without, supporting the potential of RGX-202 to drive functional improvements in patients with Duchenne Muscular Dystrophy.

，结果显示，带有CT结构域的微营养不良蛋白相比没有CT结构域的微营养不良蛋白提升了功能效益，这支持了RGX-202在杜氏肌营养不良症患者中促进功能改善的潜力。

RGX-202 is the only investigational or approved microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy (Duchenne) that includes the CT domain, a key portion of dystrophin, making it the closest to naturally occurring dystrophin.

RGX-202 是唯一一种包含 CT 结构域（抗肌萎缩蛋白的关键部分）的在研或获批的微型抗肌萎缩蛋白基因疗法候选药物，用于治疗杜氏肌营养不良症 (Duchenne)，使其最接近天然存在的抗肌萎缩蛋白。

'We specifically designed RGX-202 differently from other gene therapies with the goal of providing improved outcomes for patients, and this research further validates the potential therapeutic advantage of adding the CT domain and its importance in preventing the muscle breakdown associated with functional decline in Duchenne,' said .

“我们特意将RGX-202设计得与其他基因疗法不同，目的是为患者提供更好的治疗效果，而这项研究进一步验证了添加CT结构域的潜在治疗优势及其在预防与杜氏肌营养不良功能衰退相关的肌肉分解中的重要性，”研究人员表示。

Olivier Danos

奥利维尔·达诺斯

, Ph.D., Chief Scientific Officer of REGENXBIO. 'The positive interim results we've seen in the Phase I/II AFFINITY DUCHENNE

博士，REGENXBIO首席科学官。“我们在I/II期AFFINITY DUCHENNE中看到的积极中期结果

trial are reinforced by this preclinical research demonstrating how the novel construct of RGX-202 protects against muscle damage and supports the potential for durable, functional benefit for patients.'

试验通过这项临床前研究得到加强，该研究展示了RGX-202的新结构如何防止肌肉损伤，并为患者带来持久的功能性益处的潜力。

'AAV gene therapy holds great promise for Duchenne, and the community is in need of treatment options that have the potential to improve function and quality of life for patients,' said

“AAV基因疗法对杜氏肌营养不良症充满希望，社区需要有可能改善患者功能和生活质量的治疗选择，”他说。

Michael Kelly

迈克尔·凯利

, Ph.D., Chief Scientific Officer, CureDuchenne. 'The CT domain is a critical part of the large DMD gene and these preclinical results highlight its role in muscle health. With the science behind the novel microdystrophin construct of RGX-202, combined with the interim safety and efficacy profile seen to date in clinic, I am very encouraged by the potential of RGX-202 to be a meaningful, differentiated gene therapy for the community.'.

博士，CureDuchenne首席科学官。“CT结构域是大型DMD基因的关键部分，这些临床前结果突显了其在肌肉健康中的作用。凭借RGX-202新型微抗肌萎缩蛋白构建体背后的科学依据，加上迄今为止在临床上观察到的中期安全性和有效性特征，我非常看好RGX-202成为一种有意义且具有差异化的基因疗法的潜力。”

In this paper, titled 'Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain,' two AAV vectors, one encoding a microdystrophin protein with the CT domain and one encoding an otherwise equivalent microdystrophin protein without the CT domain, were evaluated across three studies in .

在本文中，题为“具有扩展C端结构域的微营养不良蛋白增强治疗潜力”，在三项研究中评估了两种AAV载体，一种编码带有CT结构域的微营养不良蛋白，另一种编码其他方面等效但不带CT结构域的微营养不良蛋白。

mdx

mice, a preclinical model of Duchenne, to measure muscle force, protein levels, and protection from contraction-induced muscle injury.

小鼠，杜氏肌营养不良症的临床前模型，用于测量肌肉力量、蛋白质水平以及防止收缩引起的肌肉损伤。

Compared to the microdystrophin without the CT domain, the microdystrophin that included the CT domain was found to be maintained at higher levels in transduced muscles, recruited the dystrophin-associated protein complex more effectively to the muscle membrane, and increased muscle force and resistance to damage in mice lacking dystrophin.

与不包含CT结构域的微营养不良蛋白相比，包含CT结构域的微营养不良蛋白在转导的肌肉中维持在较高水平，更有效地将营养不良蛋白相关蛋白复合物募集到肌肉膜，并增加了缺乏营养不良蛋白的小鼠的肌肉力量和抗损伤能力。

These are key factors in supporting the preservation of muscle health, as muscle damage leads to disease progression in Duchenne..

这些是支持肌肉健康 preservation 的关键因素，因为肌肉损伤会导致 Duchenne 病情进展。

These findings indicate that incorporation of the CT domain enhances the microdystrophin design by allowing for higher levels of microdystrophin to accumulate in the muscle – primarily attributed to the longer half-life of the extended microdystrophin – and may improve the functional benefit of microdystrophin gene replacement.

这些发现表明，CT结构域的加入通过允许更多的微抗肌萎缩蛋白在肌肉中积累（主要归因于延长的微抗肌萎缩蛋白更长的半衰期）改进了微抗肌萎缩蛋白的设计，并可能提升微抗肌萎缩蛋白基因替代的功能效益。

Interim results from the Phase I/II AFFINITY DUCHENNE clinical trial of RGX-202 .

AFFINITY DUCHENNE 临床试验的 I/II 期中期结果，涉及 RGX-202。

reported in

据报道在

June 2025

2025年6月

show that RGX-202 demonstrated consistent evidence of positively changing the disease trajectory of patients with Duchenne and a favorable safety profile.

表明RGX-202展示了对杜氏肌营养不良患者的疾病进程产生积极变化的一致证据，并具有良好的安全性。

REGENXBIO is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202 and expects to submit a Biologics License Application (BLA) using the accelerated approval pathway in mid-2026.

REGENXBIO 正在招募参与者参与 RGX-202 的 I/II/III 期 AFFINITY DUCHENNE 试验的关键部分，并预计将在 2026 年年中通过加速审批途径提交生物制品许可申请 (BLA)。

About RGX-202

关于RGX-202

RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain..

RGX-202 是一种潜在的最佳同类基因疗法，旨在改善杜氏肌营养不良症的功能和疗效。 RGX-202 是唯一获批或处于杜氏肌营养不良症晚期开发阶段的基因疗法，其独特的微抗肌萎缩蛋白构建体编码天然存在的抗肌萎缩蛋白的关键区域，包括 C 端 (CT) 结构域。

Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV

诸如密码子优化等额外的设计特点可能会提高基因表达，增加蛋白质翻译效率并降低免疫原性。RGX-202旨在通过NAV支持微型抗肌萎缩蛋白在骨骼肌和心肌中的递送和靶向表达。

AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress

AAV8载体和一个特性明确的肌肉特异性启动子（Spc5-12）。RGX-202由REGENXBIO使用其专有的高产量NAVXpress生产。

suspension-based platform process.

基于悬浮的平台工艺。

About Duchenne Muscular Dystrophy

关于杜氏肌营养不良症

Duchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death..

杜氏肌营养不良症是一种严重的、进行性的、退行性肌肉疾病，每年全世界每3500至5000名新生男孩中就有一人受到影响。杜氏肌营养不良症是由杜氏基因突变引起的，该基因编码一种参与肌肉细胞结构和信号通路的蛋白质——抗肌萎缩蛋白。没有抗肌萎缩蛋白，全身的肌肉会退化并变得无力，最终导致运动和独立能力的丧失，需要呼吸支持，出现心肌病并早逝。

ABOUT REGENXBIO Inc.

关于REGENXBIO公司

REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie.

REGENXBIO是一家生物技术公司，致力于通过基因治疗的治愈潜力改善生活。自2009年成立以来，REGENXBIO一直是AAV基因治疗领域的开拓者。REGENXBIO正在推进一系列针对罕见病和视网膜疾病的晚期一次性治疗管线，包括用于治疗杜氏肌营养不良的RGX-202；与日本新药株式会社合作的用于治疗MPS II的clemidsogene lanparvovec（RGX-121）和用于治疗MPS I的RGX-111；以及与艾伯维合作的用于治疗湿性AMD和糖尿病视网膜病变的surabgene lomparvovec（ABBV-RGX-314）。

Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit .

成千上万名患者已经通过 REGENXBIO 的 AAV 平台接受了治疗，其中包括接受诺华公司 ZOLGENSMA® 治疗的患者。REGENXBIO 的在研基因疗法有潜力改变数百万人的医疗保健服务方式。欲了解更多信息，请访问。

www.regenxbio.com

。

FORWARD-LOOKING STATEMENTS

前瞻性声明

This press release includes 'forward-looking statements,' within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as 'believe,' 'may,' 'will,' 'estimate,' 'continue,' 'anticipate,' 'assume,' 'design,' 'intend,' 'expect,' 'could,' 'plan,' 'potential,' 'predict,' 'seek,' 'should,' 'would' or by variations of such words or by similar expressions.

本新闻稿包含《1933年证券法》第27A条（经修订）和《1934年证券交易法》第21E条（经修订）所指的“前瞻性陈述”。这些陈述表达了一种信念、期望或意图，并通常伴随着传达预计未来事件或结果的词语，例如“相信”、“可能”、“将”、“估计”、“继续”、“预期”、“假设”、“设计”、“打算”、“预计”、“能够”、“计划”、“潜力”、“预测”、“寻求”、“应该”、“会”，或此类词语的变体或类似表述。

The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances.

前瞻性声明包括与 REGENXBIO 未来的运营和临床试验相关的声明。REGENXBIO 已基于其当前的预期、假设，以及根据其经验、对历史趋势、当前状况和预期未来发展的看法以及其他 REGENXBIO 认为在当前情况下适当的因素所作出的分析，提出了这些前瞻性声明。

However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and.

然而，实际结果和发展是否会符合REGENXBIO的预期和预测，仍受到许多风险和不确定因素的影响，包括REGENXBIO及其被许可方和合作伙伴进行的临床试验的入组、启动和完成时间及成功与否、新产品的及时开发与上市、获得并维持产品候选物的监管批准的能力、获得并维持产品候选物和技术的知识产权保护的能力、REGENXBIO所处业务和市场的趋势与挑战、产品候选物潜在市场的规模与增长以及服务这些市场的能力、速度和程度。

December 31, 2024

2024年12月31日

, and comparable 'risk factors' sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at

，以及REGENXBIO的10-Q表季度报告和其他提交给美国证券交易委员会（SEC）的文件中可比较的“风险因素”部分，这些文件已提交至SEC，并可在SEC的网站上查阅。

WWW.SEC.GOV

. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations.

本新闻稿中所有的前瞻性声明均明确受到本文中包含或提及的警示性声明的限制。预期的实际结果或发展可能无法实现，或者即使基本实现，也可能不会对REGENXBIO及其业务或运营产生预期的影响或效果。

Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release.

此类声明并非对未来业绩的保证，实际结果或发展可能与前瞻性声明中预测的内容存在重大差异。读者谨记，不要过分依赖本新闻稿中包含的前瞻性声明。这些前瞻性声明仅截至本新闻稿发布之日有效。

Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise..

除非法律要求，REGENXBIO不承担任何义务，并明确拒绝任何更新或修改任何前瞻性声明的义务，无论是否由于新信息、未来事件或其他原因。

Zolgensma

is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO.

是诺华公司注册商标。本文提到的所有其他商标均为REGENXBIO的注册商标。

Contacts:

联系人：

Dana Cormack

达娜·科马克

Corporate Communications

企业传播

dcormack@regenxbio.com

Investors: