-Preclinical findings show exosome-based therapeutic approach for the treatment of arginase-1 deficiency (ARG1-D)-

-临床前研究结果显示，基于外泌体的治疗方法可治疗精氨酸酶-1缺乏症（ARG1-D）-

-Presentation to be featured in an oral session being held on May 9, 2024-

-演讲将于2024年5月9日举行的口头会议上进行-

SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024.

2024年5月9日，圣地亚哥（环球通讯社）--Capricor Therapeutics（NASDAQ:CAPR）是一家开发用于治疗罕见疾病的转化细胞和外泌体疗法的生物技术公司，今天宣布，一份摘要以新的临床前数据为特色，突出了其StealthX™外泌体平台技术的治疗潜力，已被选为2024年5月7日至11日在马里兰州巴尔的摩举行的ASGCT第27届年会的口头报告。

The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells..

这些发现突出了一种潜在的基于外泌体的方法来治疗精氨酸酶-1缺乏症（ARG1-D），这是一种罕见的遗传代谢疾病，其特征是肝脏和红细胞中完全或部分缺乏精氨酸酶。。

“The findings from this preclinical study have further characterized our StealthX™ exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We believe our StealthX™ platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads.

Capricor首席执行官Linda Marbán博士说：“这项临床前研究的结果进一步表征了我们的StealthX™外泌体平台，并为基于外泌体的酶替代疗法提供了一种新方法，旨在替代缺乏或缺失的酶。”。“我们相信我们的StealthX™平台为潜在的广泛应用提供了机会，我们的数据继续支持外泌体作为各种有效载荷的合适运载工具的概念。

We continue to focus our exosome platform in two main areas, the development of therapeutics and vaccines, and our goal is to leverage partnership opportunities to further expand and advance these opportunities.”.

我们继续将外泌体平台的重点放在两个主要领域，即治疗和疫苗的开发，我们的目标是利用合作机会进一步扩大和推进这些机会。”。

Study Overview and Findings:

研究概述和发现：

In this study, exosomes were engineered to express human Arg1 enzyme inside of the exosomes and were evaluated for their in vitro functionality as an approach for the potential treatment of ARG1-D.

在这项研究中，外泌体被设计为在外泌体内部表达人Arg1酶，并评估其体外功能，作为潜在治疗Arg1-D的方法。

Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro. In addition, the Arg1-exosomes were capable of delivering the Arg1 protein into 293F and HepG2 cells in a time and dose-dependent manner, contrary to human recombinant Arg1 protein alone at the same or higher dose..

结果表明，Arg1外泌体具有酶活性，能够在体外将精氨酸转化为尿素。此外，与单独使用相同或更高剂量的人重组Arg1蛋白相反，Arg1外泌体能够以时间和剂量依赖性方式将Arg1蛋白递送至293F和HepG2细胞。。

The data suggests that the Arg1-exosomes were able to not only catalyze arginine efficiently at a lower delivered dose to cells in vitro but also protect the Arg1 protein to be delivered into target cells and retain its enzymatic activity in cells.

数据表明，Arg1外泌体不仅能够在体外以较低的递送剂量有效催化精氨酸，而且能够保护Arg1蛋白被递送到靶细胞中并在细胞中保持其酶活性。

Taken together, Capricor’s StealthX™ platform of engineered Arg1-exosomes as tested in vitro have the potential to serve as an enzyme replacement therapy to deliver Arg1 to hepatocytes and possibly have potential clinical benefits for the treatment of ARG1-D.

综上所述，经体外测试，Capricor的StealthX™工程化Arg1外泌体平台有可能作为酶替代疗法将Arg1递送至肝细胞，并可能对Arg1-D的治疗具有潜在的临床益处。

Presentation details:

演示详细信息：

Title:

标题：

Exosome-mediated intracellular delivery of arginase-1: a potential application for the treatment of arginase-1 deficiency

外泌体介导的精氨酸酶-1的细胞内递送：治疗精氨酸酶-1缺乏症的潜在应用

Oral session:

口头会议：

Exosomes, Virus like Particles and LNPs

外来体，病毒样颗粒和LNP

Presenting author:

演示作者：

Li-En Hsieh, Ph.D., Principal Scientist

李恩谢博士，首席科学家

Date:

日期：

May 9, 2024 at 4:45 p.m. ET

2024年5月9日，美国东部时间下午4:45

Abstract number:

摘要编号：

223

223

The abstract will be made available on the publications section of the Capricor website following the conclusion of the meeting.

会议结束后，将在Capricor网站的出版物部分提供摘要。

About Capricor Therapeutics

关于Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, CAP-1002 — an allogeneic cardiac-derived cell therapy.

Capricor Therapeutics，Inc.（纳斯达克股票代码：CAPR）是一家生物技术公司，致力于推进基于转化细胞和外泌体的疗法，以重新定义罕见疾病的治疗前景。我们创新的最前沿是我们的主要候选产品CAP-1002-一种同种异体心脏衍生细胞疗法。

Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD).

广泛的临床前和临床研究表明，CAP-1002可以证明专门针对肌营养不良症和心脏病的免疫调节，抗纤维化和再生作用。CAP-1002目前正在进行治疗杜兴氏肌营养不良症（DMD）的3期临床开发。

Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases.

Capricor还利用我们的外泌体技术的力量，在临床前开发中使用我们专有的StealthX™平台，专注于疫苗学领域，靶向递送寡核苷酸，蛋白质和小分子疗法，以潜在地治疗和预防各种疾病。

At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter..

在Capricor，我们致力于突破可能性的界限，为有需要的人开辟一条变革性治疗之路。有关更多信息，请访问capricor.com，并在Facebook、Instagram和Twitter上关注capricor。。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性声明的警示说明

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

本新闻稿中关于Capricor候选产品的功效，安全性和预期用途的声明；发现工作和临床试验的开始，行为，规模，时间和结果；临床试验的注册速度；关于监管文件，未来研究和临床试验的计划；涉及产品的监管发展，包括获得监管批准或以其他方式将产品推向市场的能力；制造能力；监管会议日期；关于我们财务前景的报表；实现产品里程碑和从商业合作伙伴处获得里程碑付款的能力；关于当前和未来合作活动以及商业权利所有权的计划；知识产权的范围、期限、有效性和可执行性；未来收入流和预测；对最近完成的发行收益的预期用途和发行的预期效果的预期；以及关于Capricor管理团队未来期望、信念、目标、计划或前景的任何其他声明，构成1995年《私人证券诉讼改革法案》含义内的前瞻性声明。

Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements.

任何不属于历史事实陈述的陈述（包括包含“相信”、“计划”、“可能”、“预期”、“期望”、“估计”、“应该”、“目标”、“将会”、“将会”和类似表述的陈述）也应视为前瞻性陈述。有许多重要因素可能导致实际结果或事件与此类前瞻性声明所示的结果或事件存在重大差异。

More information about these and other risks that may impact Capricor’s business is set fo.

有关这些风险和其他可能影响Capricor业务的风险的更多信息，请参见。

Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. CAP-1002 is an Investigational New Drug and is not approved for any indications.

Capricor已与日本新屋株式会社（美国子公司：NS Pharma，Inc.）签订合作伙伴关系，在美国和日本独家商业化和分销DMD CAP-1002。CAP-1002是一种研究性新药，未被批准用于任何适应症。

None of Capricor’s exosome-based candidates have been approved for clinical investigation..

Capricor的基于外泌体的候选人均未被批准用于临床研究。。

For more information, please contact:

有关更多信息，请联系：

Capricor Company Contact:

Capricor公司联系人：

AJ Bergmann, Chief Financial Officer

首席财务官AJ Bergmann

abergmann@capricor.com

abergmann@capricor.com

858.727.1755

858.727.1755