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Capricor Therapeutics to Present Exosome Platform Updates at 2024 American Association of Extracellular Vesicles Annual Meeting

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Capricor Therapeutics (NASDAQ: CAPR) announced the presentation of new preclinical data on their StealthX™ exosome platform at the 2024 American Association of Extracellular Vesicles Annual Meeting in Houston. The research showcases an innovative exosome-based approach for treating Duchenne muscular dystrophy (DMD) through the delivery of phosphorodiamidate morpholino oligomers (PMOs).

The study demonstrates improved cellular uptake and enhanced exon skipping efficiency using exosome-based therapeutics. CEO Linda Marbán highlighted the platform's potential as a delivery vehicle for various payloads and expressed interest in pursuing partnership opportunities to advance the technology.

Capricor Therapeutics (NASDAQ: CAPR) ha annunciato la presentazione di nuovi dati preclinici sulla loro piattaforma di esosomi StealthX™ durante il Meeting Annuale dell'American Association of Extracellular Vesicles 2024 a Houston. La ricerca presenta un approccio innovativo basato sugli esosomi per il trattamento della distrofia muscolare di Duchenne (DMD) attraverso la somministrazione di oligomeri morfolinici fosforodiamidati (PMO).

Lo studio dimostra un miglioramento dell'assorbimento cellulare e un'efficienza aumentata nel salto degli esoni utilizzando terapie basate sugli esosomi. Il CEO Linda Marbán ha sottolineato il potenziale della piattaforma come veicolo di consegna per vari carichi utili ed ha espresso interesse nel perseguire opportunità di partnership per far progredire la tecnologia.

Capricor Therapeutics (NASDAQ: CAPR) anunció la presentación de nuevos datos preclínicos sobre su plataforma de exosomas StealthX™ en la Reunión Anual de la Asociación Americana de Vesículas Extracelulares 2024 en Houston. La investigación muestra un enfoque innovador basado en exosomas para tratar la mediante la entrega de oligómeros morfolinicos fosforodiamidatos (PMOs).

El estudio demuestra una mejora en la captación celular y una mayor eficiencia en el salto de exones utilizando terapias basadas en exosomas. La CEO Linda Marbán destacó el potencial de la plataforma como vehículo de entrega para diversas cargas y expresó interés en perseguir oportunidades de asociación para avanzar en la tecnología.

Capricor Therapeutics (NASDAQ: CAPR)는 휴스턴에서 열리는 2024년 미국 세포외 소포 협회 연례 회의에서 StealthX™ 엑소좀이 플랫폼에 대한 새로운 전임상 데이터를 발표했습니다. 이 연구는 두셴 근육 이영양증 (DMD) 치료를 위한 엑소좀 기반 접근 방식을 통해 인산다이아미드 모폴리노 올리고머(PMO)를 전달하는 혁신적인 방법을 보여줍니다.

이 연구는 엑소좀 기반 치료제를 사용하여 세포 흡수 향상과 엑손 건너뛰기 효율성을 개선했음을 입증합니다. CEO인 린다 마르반은 다양한 유효 성분에 대한 전달 수단으로서 플랫폼의 잠재력을 강조하고 이 기술을 발전시키기 위해 파트너십 기회를 모색하겠다고 밝혔습니다.

Capricor Therapeutics (NASDAQ: CAPR) a annoncé la présentation de nouvelles données précliniques sur sa plateforme d'exosomes StealthX™ lors de la Réunion Annuelle de l'Association Américaine des Vesicules Extracellulaires 2024 à Houston. La recherche met en avant une approche novatrice basée sur des exosomes pour traiter la dystrophie musculaire de Duchenne (DMD) via la livraison d'oligomères morpholino phosphorodiamidés (PMOs).

L'étude démontre une meilleure absorption cellulaire et une efficacité accrue du saut d'exons grâce aux thérapies basées sur les exosomes. La PDG Linda Marbán a souligné le potentiel de la plateforme en tant que véhicule de livraison pour divers porteurs et a exprimé son intérêt pour rechercher des opportunités de partenariat afin de faire avancer la technologie.

Capricor Therapeutics (NASDAQ: CAPR) gab die Präsentation neuer präklinischer Daten über ihre StealthX™ Exosomenplattform auf dem Treffen der American Association of Extracellular Vesicles 2024 in Houston bekannt. Die Forschung zeigt einen innovativen, auf Exosomen basierenden Ansatz zur Behandlung der Duchenne-Muskeldystrophie (DMD) durch die Lieferung von phosphordiami-dat-morpholino-Oligomeren (PMOs).

Die Studie zeigt eine verbesserte zelluläre Aufnahme und eine erhöhte Effizienz beim Exonspringen unter Verwendung von auf Exosomen basierenden Therapeutika. CEO Linda Marbán hob das Potenzial der Plattform als Transportmittel für verschiedene Wirkstoffe hervor und äußerte Interesse an Partnerschaftsmöglichkeiten zur Weiterentwicklung der Technologie.

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-Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-

SAN DIEGO, Nov. 12, 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 is being presented at the 2024 American Association of Extracellular Vesicles Annual Meeting taking place in Houston, Texas from November 10-13, 2024. The findings highlight a potential exosome-based approach for the treatment of Duchenne muscular dystrophy (DMD) by delivering phosphorodiamidate morpholino oligomers (PMOs).

“We continue to build and characterize our StealthX™ exosome platform and this study further supports that endeavor as we provide a novel approach for exosome-based PMO delivery for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This study further demonstrates the potential for an exosome-based therapeutic approach to exhibit better cellular uptake and promote higher exon skipping efficiency. Furthermore, our platform continues to support the concept of exosomes as a potential suitable delivery vehicle for a variety of payloads and we continue to support our goal to leverage partnership opportunities to further advance this technology.”

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

About 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, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its 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. 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.

Cautionary Note Regarding Forward-Looking Statements

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; potential future agreements; 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. 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. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as filed with the Securities and Exchange Commission on August 8, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (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. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755


FAQ

What is Capricor Therapeutics presenting at the 2024 AAEV Annual Meeting?

Capricor Therapeutics (CAPR) is presenting new preclinical data on their StealthX™ exosome platform technology, specifically focusing on exosome-based delivery of PMOs for treating Duchenne muscular dystrophy.

How does Capricor's StealthX platform aim to treat DMD?

The StealthX platform uses exosome-based delivery of phosphorodiamidate morpholino oligomers (PMOs), showing improved cellular uptake and higher exon skipping efficiency in treating Duchenne muscular dystrophy.

When and where is Capricor presenting their exosome platform updates?

Capricor is presenting at the 2024 American Association of Extracellular Vesicles Annual Meeting in Houston, Texas, taking place from November 10-13, 2024.

What are the key benefits of Capricor's exosome-based therapeutic approach?

The approach demonstrates better cellular uptake, promotes higher exon skipping efficiency, and shows potential as a suitable delivery vehicle for various therapeutic payloads.

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