Exosomes are nano-sized, membrane-enclosed vesicles that are secreted by essentially all cells and contain bioactive molecules, including proteins, RNAs and microRNAs. They act as messengers to regulate the functions of neighboring cells and pre-clinical research has shown that exogenously-administered exosomes can direct or, in some cases, re-direct cellular activity, thereby supporting their therapeutic potential. Their size, ease of crossing cell membranes, and ability to communicate in native cellular language makes them an exciting, emerging class of potential therapeutic agents.
Our exosomes program consists of exosomes derived from the company’s proprietary cardiosphere-derived cells CDCs and engineered exosomes, both of which are in various stages of preclinical development. While CAP-2003 was the initial technology used in preclinical development, we have expanded Capricor’s pipeline to include additional exosomes technologies.
We are now focused on developing a precision-engineered exosome platform technology that can carry defined sets of effector molecules that exert their effects through defined mechanisms of action. We have announced the planned expansion of our exosome platform technology that potentially may be used for vaccine development, vesicle-mediated protein therapies and treatment of inherited diseases.
Exosomes act as messengers to regulate the functions of neighboring or distant cells and have been shown to regulate functions such as cell survival, proliferation, inflammation, and tissue regeneration. Furthermore, pre-clinical research has shown that exogenously-administered exosomes can modify cellular activities, thereby supporting their therapeutic potential. Their size, low or null immunogenicity, and ability to communicate in native cellular language makes them an exciting new class of therapeutic agents with the potential to expand our ability to address complex biological responses. Because exosomes are a cell-free substance, they can be stored, handled, reconstituted and administered in a similar fashion to common biopharmaceutical products such as antibodies.
We have promising pre-clinical data in several indications from studies done in our labs as well as in collaboration with other academic institutions. Additionally, in July 2018, we entered into a Cooperative Research and Development Agreement with the U.S. Army Institute of Surgical Research (USAISR) pursuant to which we agreed to cooperate in research and development on the evaluation of our CDC-exosomes for the treatment of trauma-related injuries and conditions which are now the third leading cause of death in the U.S.
Capricor has exclusively licensed intellectual property relating to CDC-exosomes from Cedars-Sinai Medical Center and is also pursuing its own intellectual property rights relating to exosome technologies.
Capricor is also developing its exosome platform as a potential vaccine for COVID-19. Click here to view a webcast by Dr. Stephen Gould from Johns Hopkins University for more information on this program.
View a list of publications relating to Capricor’s exosome technology.