Capricor Therapeutics Presents New Data Demonstrating a Scalable Framework for Loading Therapeutic Oligonucleotides into Exosomes at AAEV 2025

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) presented new preclinical data at AAEV 2025 (Nov 20–23, 2025) describing a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes.

Key findings: engineered exosomes from 293F cells were loaded with siRNA and PMO using optimized electroporation; both scale-up and scale-out electroporation produced comparable loading efficiencies to small-volume electroporation; integrating the two approaches enabled manufacturing of significantly larger batches of loaded exosomes, offering a pathway to produce clinically relevant quantities to support future clinical development.

Insights

Exosome Manufacturing Expert

Capricor demonstrates a reproducible, scalable electroporation framework for loading siRNA and PMO into exosomes.

Engineered exosomes from 293F cells were loaded with siRNA and PMO using optimized electroporation, and both scale-up and scale-out approaches matched small-volume efficiencies.

The business mechanism links an operationally scalable loading process to the ability to produce larger batch volumes, which is a direct enabler for advancing into later-stage clinical work and larger preclinical studies.

Key dependencies include reproducible batch-to-batch yields, maintained cargo integrity after scale, and validated process controls for manufacturing; these are necessary to move from poster data to regulated production.

Watch for published quantitative loading yields, validation of potency post-loading, and any announced process validation or GMP transfer over the next 12–24 months.

Translational Development Expert

Scalable loading of oligonucleotides into exosomes removes a key manufacturing hurdle for clinical translation.

The framework claims comparable loading efficiency for scale-up and scale-out electroporation and integration of both enables substantially larger batch production for clinical needs.

Risks remain around confirming that scaled material meets safety, potency, and regulatory standards; reproducible functional delivery in relevant models must be shown before clinical application.

Concrete items to monitor include formal potency assays on loaded exosomes, regulatory interactions about manufacturing, and any announcements tied to advancing exosome candidates into formal clinical programs within 12–36 months.

--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
rcona@kcsa.com
212.896.1204

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

FAQ

What did Capricor (CAPR) present at AAEV 2025 about exosome loading?

Capricor presented a scalable framework showing optimized electroporation can load siRNA and PMO into 293F-derived exosomes with comparable efficiencies at larger scales.

How could Capricor's scalable loading affect CAPR's clinical development timeline?

The company said the framework provides a feasible pathway to produce clinically relevant quantities of loaded exosomes to support future clinical development.

Which cargo types did Capricor demonstrate loading into exosomes in the AAEV 2025 poster?

The poster demonstrated loading of small interfering RNA (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into engineered exosomes.

What manufacturing strategies did Capricor report for scaling exosome loading (CAPR)?

Capricor reported both scale-up and scale-out electroporation strategies and noted that integrating them enables significantly larger batch manufacturing.

When and where was Capricor's exosome loading data presented for CAPR?

The data were presented at the AAEV 2025 Annual Meeting in Salt Lake City, Utah, held November 20–23, 2025.