New Data Demonstrates Exosome-Mediated Delivery of mRNAs With Enhanced Expression and Lower Toxicity Compared to Lipid Nanoparticles
- Functional Enzyme Expression and Real-Time Imaging of mRNA Expression in Live Animals-
- Platform for Advancing Clinical Development of Exosome-RNA Based Vaccine Candidate-
- Data to be Discussed on Earnings Call this Thursday, March 11 at 4:30 p.m. ET-
Capricor Therapeutics, a biotechnology company focused on the development of transformative cell- and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, in collaboration with researchers, announced today that new advances from its positive preclinical study for a multivalent exosome-based mRNA vaccine for COVID-19 have been posted on the bioRxiv preprint server and has been submitted for publication.
“Capricor previously demonstrated that our exosome-based multivalent RNA delivery platform can induce long-lasting immune responses to multiple SARS-CoV-2 proteins, and potentially elicit a broad-based, cellular and humoral immunity. We decided to explore further the expression of RNAs with our exosome-based delivery system and compare exosomes with lipid nanoparticles specifically focused on short term toxicity,” said Linda Marbán, Ph.D., CEO of Capricor. “The data demonstrated functional RNA expression in vivo further showing the power of our exosome platform to potentially expand into areas beyond SARS-CoV-2. This strengthens my belief that our exosome platform can deliver RNA effectively into cells and drive the expression of functional proteins. The opportunities for pipeline expansion are very exciting, both in vaccines and in the delivery of therapeutic RNAs.”
Key takeaways from this pre-clinical study include:
- Real-time imaging establishing exosome-mediated mRNA expression in living animals
- Demonstration that exosome-mRNA formulations can drive functional enzyme expression in vivo, opening the opportunity for therapeutic exosome-mRNA formulations
- Superior expression from exosome-mRNA formulations compared to Lipid Nanoparticle (LNP)-mRNA formulations
- Exosome-mRNA treatment and exosome injections are free of adverse effects, in contrast to LNP-mRNA treatments and LNP injections
Exosomes are the body’s own drug delivery vehicle, produced by all cells, abundant in all biofluids, and demonstrated to be safe by decades of transfusion and transplantation medicine. Their small size, biological origin, minimal immunogenicity and normal role in delivering signals and RNAs to human cells, indicates that they have the potential to expand the range of therapeutics that can be deployed in the fight against a broad spectrum of diseases.
Dr. Marbán continued, “This data is of great importance to Capricor, because it demonstrates the efficacy of our vaccine candidate in mice, as well as corroborates functional RNA expression and demonstrates a potentially safer delivery vehicle compared to commonly used LNPs. We are planning to meet with the U.S. Food and Drug Administration this quarter to discuss next steps for a clinical development strategy for our exosome-mRNA vaccine and look forward to sharing additional updates as they become available.”
Conference Call and Webcast Details
Capricor’s Executive Consultant, Dr. Stephen Gould, and Capricor’s management will host a conference call on Thursday, March 11, 2021 to discuss this data and provide a corporate update. To participate, please dial 877-451-6152 (Domestic/Toll-Free) or 201-389-0879 (International) and reference the conference ID: 13717080
To participate via a webcast (with slides), please visit: http://public.viavid.com/index.php?id=143779.
The webcast will be archived for approximately 30 days and will be available at: http://capricor.com/news/events/.
Source – Capricor Therapeutics