November 24, 2025 — Leads & Copy —
SAN DIEGO, Nov. 24, 2025 – Capricor Therapeutics, Inc. (NASDAQ: CAPR) has announced new data outlining a scalable framework for loading therapeutic small interfering RNAs (siRNA) and phosphorodiamidate morpholino oligomers (PMO) into exosomes. The findings support the versatility of Capricor’s exosome technology, enabling efficient and scalable loading of siRNAs and PMOs to support future clinical development.
The data was presented at the 2025 American Association for Extracellular Vesicles (AAEV) Annual Meeting in Salt Lake City, Utah, which took place from November 20-23, 2025. The poster, titled “A Systematic Framework for the Scalable Loading of Therapeutic siRNA and PMO into Exosomes,” showcased proprietary data highlighting Capricor’s exosome-based technology. It provided an overview of scale-up and scale-out electroporation strategies that can be integrated to achieve substantially larger yields of exogenously loaded engineered exosomes, a critical requirement for further clinical development.
Key findings from the presentation included:
- 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.
Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics, said the presentation at AAEV 2025 underscores the strength of Capricor’s exosome platform by demonstrating ongoing scientific advancement and the relevance of exosome-based delivery for the potential treatment of a broad range of diseases. Establishing a scalable, reproducible loading process is essential as Capricor continues to advance its exosome pipeline for therapeutic development, Marbán added.
Capricor Therapeutics (NASDAQ: CAPR) is focused on advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. Its lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage clinical development for the treatment of Duchenne muscular dystrophy (DMD). Preclinical and clinical data have demonstrated Deramiocel’s immunomodulatory and anti-fibrotic effects in helping to preserve cardiac and skeletal muscle function in DMD.
Capricor is also leveraging 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.
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.
Raquel Cona, KCSA Strategic Communications, can be reached at rcona@kcsa.com or 212.896.1204.
AJ Bergmann, Chief Financial Officer of Capricor, can be reached at abergmann@capricor.com or 858.727.1755.
Source: Capricor Therapeutics
