Capricor Therapeutics Presents New Data Demonstrating a Scalable Framework for Loading Therapeutic Oligonucleotides into Exosomes at AAEV 2025
--Findings support the versatility of Capricor's exosome technology, enabling efficient and scalable loading of siRNAs and PMOs to support future clinical development--
SAN DIEGO, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc., (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics, today announced new data describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes. The data were presented at the 2025 American Association for Extracellular Vesicles (AAEV) Annual Meeting in Salt Lake City, Utah, held from November 20-23, 2025.
The poster titled, "A Systematic Framework for the Scalable Loading of Therapeutic siRNA and PMO into Exosomes," highlighted proprietary data showcasing Capricor's exosome-based technology. The data provided an overview of both scale-up and scale-out electroporation strategies that can be integrated to achieve substantially larger yields of exogenously loaded engineered exosomes, which is a critical requirement for further clinical development.
Key findings include:
- Engineered exosomes derived from 293F cells were successfully loaded with therapeutic siRNA and PMO cargo using optimized electroporation conditions.
- Both scale-up and scale-out strategies produced comparable loading efficiencies to standard small-volume electroporation.
- Integrating the two approaches enables manufacturing of significantly larger batches of therapeutic exosomes.
- The framework provides a feasible pathway for producing clinically relevant quantities of loaded exosomes to support future clinical development.
"Presenting this work at AAEV 2025 underscores the strength of our exosome platform by demonstrating ongoing scientific advancement and the relevance of exosome-based delivery for the potential treatment of a broad range of diseases," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics. "Establishing a scalable, reproducible loading process is essential as we continue to advance our exosome pipeline for therapeutic development. As we continue to grow this platform, we remain focused on disciplined execution and building long-term value for both patients and shareholders alike."
View the AAEV 2025 poster presentation here.
About Capricor Therapeutics
Capricor Therapeutics (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, an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD). Extensive preclinical and clinical data have demonstrated Deramiocel's potent immunomodulatory and anti-fibrotic effects in helping to preserve cardiac and skeletal muscle function in DMD. Capricor is also leveraging the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics, with the potential to treat and prevent a wide range of diseases. At Capricor, we are 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 X.
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 clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; 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, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, as filed with the Securities and Exchange Commission on November 10, 2025. 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 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
[email protected]
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
[email protected]
858.727.1755
