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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.

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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.