A single ABBV-RGX-314 gene therapy treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing wet AMD due to the treatment burden of chronic anti-VEGF injections

通过长期维持视力健康并克服由于慢性抗VEGF注射的治疗负担而导致的湿性AMD管理的临床挑战，单一的ABBV-RGX-314基因治疗疗法有可能成为抗VEGF治疗中新的护理标准选择

Patients who received therapeutic doses resulted in stable or improved vision and retinal anatomy up to 2 years

接受治疗剂量的患者可以稳定或改善视力和视网膜解剖长达2年

Additional long-term follow-up data has demonstrated durable treatment effect, with stable or improved vision, up to 4 years

其他长期随访数据表明，治疗效果持久，视力稳定或改善，长达4年

Enrollment is on track in pivotal trials of ABBV-RGX-314 in wet AMD that are expected to support global regulatory submissions in late 2025 through the first half of 2026

ABBV-RGX-314在湿性AMD中的关键试验正在进行中，预计将在2025年末至2026年上半年支持全球监管提交

ROCKVILLE, Md., March 28, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of results from the Phase I/IIa trial evaluating the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD).  Two-year data were published in The Lancet in a paper titled 'Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study.' These positive study results informed the ongoing pivotal trials of ABBV-RGX-314, a potential one-time gene therapy, for the treatment of wet AMD..

ROCKVILLE，Md.，2024年3月28日/PRNewswire/--REGENXBIO Inc.（纳斯达克：RGNX）今天宣布公布了I/IIa期试验的结果，该试验评估了单剂量视网膜下ABBV-RGX-314治疗湿性年龄相关性黄斑变性（湿性AMD）的安全性和耐受性。两年的数据发表在《柳叶刀》上，发表在一篇题为“视网膜下递送RGX-314治疗新生血管性年龄相关性黄斑变性的基因治疗：1/2a期剂量递增研究”的论文中这些积极的研究结果为正在进行的ABBV-RGX-314（一种潜在的一次性基因疗法）治疗湿性AMD的关键试验提供了依据。。

'We have started 2024 with strong, positive new data from the ABBV-RGX-314 program, and we believe that there is multi-billion-dollar potential for ABBV-RGX-314 to become a first-in-class gene therapy for wet AMD and the standard of care to treat and prevent progression of diabetic retinopathy,' said Kenneth T.

KennethT说：“我们已经从2024年开始使用来自ABBV-RGX-314计划的强大而积极的新数据，我们相信ABBV-RGX-314有数十亿美元的潜力成为湿性AMD的一流基因疗法，以及治疗和预防糖尿病视网膜病变进展的护理标准。”。

Mills, President and Chief Executive Officer of REGENXBIO. 'To have these Phase I/IIa data published in The Lancet highlights the groundbreaking work of our scientists and investigators, and further validates the clinically transformative nature of ABBV-RGX-314 as a potential one-time gene therapy for wet AMD that may help patients maintain or improve their vision.'.

REGENXBIO总裁兼首席执行官米尔斯说：“将这些I/IIa期数据发表在《柳叶刀》上，突出了我们科学家和研究人员的开创性工作，并进一步验证了ABBV-RGX-314作为湿性AMD潜在的一次性基因治疗的临床转化性质，可能有助于患者维持或改善视力。”。

The findings highlighted in The Lancet demonstrated that a single administration of ABBV-RGX-314 was generally well tolerated. Stable or improved visual acuity and retinal thickness was observed with few or no supplemental anti-VEGF injections in most patients at two years. Patients who received therapeutic doses demonstrated sustained levels of ABBV-RGX-314 protein and stable or improved vision and retinal anatomy with few, to no, supplemental anti-VEGF injections in most participants up to two years.

《柳叶刀》强调的研究结果表明，单次服用ABBV-RGX-314通常耐受性良好。大多数患者在两年内很少或没有补充抗VEGF注射，观察到稳定或改善的视力和视网膜厚度。接受治疗剂量的患者表现出持续的ABBV-RGX-314蛋白水平，视力和视网膜解剖结构稳定或改善，大多数参与者在两年内几乎没有补充抗VEGF注射。

REGENXBIO has also reported additional positive interim data from a long-term follow-up study of ABBV-RGX-314 supporting that treatment continues to be well-tolerated and demonstrates long-term, durable treatment effect up to four years..

REGENXBIO还报告了来自ABBV-RGX-314长期随访研究的额外积极中期数据，该研究支持治疗继续具有良好的耐受性，并显示出长达四年的长期持久治疗效果。。

'The publication of the ABBV-RGX-314 Phase I/IIa trial results in The Lancet reinforces the encouraging long-term clinical data observed using subretinal delivery and underscores the potential of ABBV-RGX-314 gene therapy to offer a new approach to the clinical management of wet AMD,' said Jeffrey S.

Jeffrey S说：“在《柳叶刀》上发表的ABBV-RGX-314 I/IIa期试验结果强化了使用视网膜下递送观察到的令人鼓舞的长期临床数据，并强调了ABBV-RGX-314基因治疗为湿性AMD的临床管理提供新方法的潜力。”。

Heier, M.D., Director of the Vitreoretinal Service and Director of Retina Research at Ophthalmic Consultants of Boston and primary investigator for the trial. 'Wet AMD is a chronic, life-long disease and real-world evidence shows patients are losing significant vision over time, and the burden of frequent anti-VEGF injections needed to manage their wet AMD is a major reason why.

黑尔（Heier，M.D.）是波士顿眼科顾问公司（Ophthalmic Consultants）玻璃体视网膜服务主任和视网膜研究主任，也是该试验的主要研究者。”湿性AMD是一种慢性终身疾病，现实世界的证据表明，随着时间的推移，患者正在失去显着的视力，而管理湿性AMD所需的频繁抗VEGF注射的负担是其主要原因。

A single treatment of ABBV-RGX-314 that can potentially provide long-lasting treatment outcomes and a strong safety profile would offer a novel approach to treating this serious and blinding disease.'.

ABBV-RGX-314的单一治疗可能会提供持久的治疗效果和强大的安全性，这将为治疗这种严重且致盲的疾病提供一种新方法。”。

ABBV-RGX-314 is currently being evaluated in patients with wet AMD in two pivotal trials called ATMOSPHERE® and ASCENT™. Enrollment is on track and these trials are expected to support global regulatory submissions with the U.S. Food and Drug Administration and the European Medicines Agency in late 2025 through the first half of 2026..

ABBV-RGX-314目前正在两项称为ATMOSPHERE®和ASCENT™的关键试验中对湿性AMD患者进行评估。招生工作正在进行中，预计这些试验将支持向美国提交的全球监管报告。S、 2025年末至2026年上半年，美国食品和药物管理局（FDA）和欧洲药品管理局（European Medicines Agency）。。

About ABBV-RGX-314ABBV-RGX-314, being developed in collaboration with AbbVie, is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. ABBV-RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF).

关于与AbbVie合作开发的ABBV-RGX-314ABBV-RGX-314，正在研究作为湿性AMD，糖尿病视网膜病变和其他慢性视网膜疾病的潜在一次性治疗方法。ABBV-RGX-314由NAV®AAV8载体组成，该载体编码旨在抑制血管内皮生长因子（VEGF）的抗体片段。

ABBV-RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina..

据信，ABBV-RGX-314抑制VEGF途径，通过该途径，新的渗漏血管生长并促进视网膜中的液体积聚。。

REGENXBIO is advancing research in two separate routes of administration of ABBV-RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc.

REGENXBIO正在通过标准化的视网膜下递送程序以及向脉络膜上空间的递送，推进两种不同途径向眼睛施用ABBV-RGX-314的研究。REGENXBIO已从Clearside Biomedical，Inc.获得SCS Microinjector®的某些专有权。

to deliver gene therapy treatments to the suprachoroidal space of the eye..

为眼脉络膜上间隙提供基因治疗。。

About Wet AMDWet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients.

关于湿性AMD AMD的特征是由于视网膜中新的渗漏血管形成而导致视力丧失。湿性AMD是美国、欧洲和日本视力丧失的重要原因，仅在这些地区就有多达200万人患有湿性AMD。目前的抗VEGF疗法已经显着改变了湿性AMD的治疗前景，由于其能够预防大多数患者视力丧失的进展，因此成为标准治疗方法。

These therapies, however, require life-long frequent, repeated intraocular injections to maintain efficacy. Due to the burden of treatment, it is difficult for patients to adhere to frequent injections, which can lead to a decline in vision over time..

然而，这些疗法需要终身频繁，反复的眼内注射才能维持疗效。由于治疗负担，患者很难坚持频繁注射，随着时间的推移，这可能导致视力下降。。

ABOUT REGENXBIO Inc.REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II.

关于REGENXBIO Inc.REGENXBIO是一家领先的临床阶段生物技术公司，旨在通过基因治疗的治疗潜力来改善生活。自2009年成立以来，REGENXBIO率先开发了AAV Therapeutics，这是一类创新的基因治疗药物。REGENXBIO正在推进一系列针对视网膜和罕见疾病的AAV治疗方法，包括用于治疗湿性AMD和糖尿病视网膜病变的ABBV-RGX-314，正在与AbbVie，RGX-202合作开发用于治疗Duchenne和RGX-121用于治疗MPS II。

Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com..

数千名患者接受了REGENXBIO的AAV治疗平台的治疗，包括诺华的ZOLGENSMA治疗脊髓性肌萎缩症儿童。AAV疗法旨在一次性治疗，有可能改变数百万人的医疗保健方式。有关更多信息，请访问www.regenxbio.com。。

FORWARD-LOOKING STATEMENTSThis 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.

前瞻性声明本新闻稿包括经修订的《1933年证券法》第27A节和经修订的《1934年证券交易法》第21E节所指的“前瞻性声明”。这些陈述表达了一种信念、期望或意图，通常伴随着表达预计未来事件或结果的词语，如“相信”、“可能”、“意志”、“估计”、“继续”、“预期”、“假设”、“设计”、“打算”、“期望”、“可能”、“计划”、“潜力”、“预测”、“寻求”、“应该”、“将会”或这些词语的变体或类似表达。

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未来运营和临床试验有关的声明。REGENXBIO根据其经验、对历史趋势、现状和预期未来发展的看法，以及REGENXBIO认为在这种情况下合适的其他因素，根据REGENXBIO目前的预期、假设和分析，做出了这些前瞻性陈述。

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 timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development 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 busine.

然而，实际结果和发展是否符合REGENXBIO的预期和预测，还存在许多风险和不确定性，包括REGENXBIO、其被许可方及其合作伙伴进行的临床试验的注册、开始和完成时间以及成功率，REGENXBIO及其开发合作伙伴进行的临床前研究的开始和完成时间以及成功率，新产品的及时开发和推出，获得和维持候选产品监管批准的能力，获得和维持候选产品知识产权保护的能力以及业务中的技术、趋势和挑战。

Zolgensma® is a registered trademark of Novartis Gene Therapies. SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Zolgensma®是诺华基因疗法的注册商标。SCS Microinjector®是Clearside Biomedical，Inc.的商标。此处引用的所有其他商标均为REGENXBIO的注册商标。

Contacts:

联系人：

Dana CormackCorporate Communicationsdcormack@regenxbio.com

Dana Cormack公司Communicationsdcormack@regenxbio.com

Investors:Chris Brinzey, ICR Westwicke339-970-2843chris.brinzey@westwicke.com

投资者：Chris Brinzey，ICRWestwicke339-970-2843chris.brinzey@westwicke.com

SOURCE REGENXBIO Inc.

来源REGENXBIO股份有限公司。