Capricor Therapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class cell- and exosome-based therapeutics for the treatment and prevention of a variety of diseases and disorders, announced today the publication, in collaboration with researchers at Cedars-Sinai Medical Center, which demonstrates the utility of pathway-enhancing culture conditions and small molecule inhibitors to retain markers of cell therapy potency. The publication titled, “Small molecule inhibitors and culture conditions enhance therapeutic cell and EV potency via activation of beta-catenin and suppression of THY1” was published in the international peer-reviewed journal, Nanomedicine: Nanotechnology, Biology and Medicine.
“This data from this publication is of significant importance to Capricor because it demonstrates our approach to enhance potency of cells by targeting select signaling pathways. The ability to manipulate cells provides read through to our exosome product candidates which are being engineered to treat specific diseases. We have dedicated the last few years at Capricor to understanding the molecular composition of our exosomes products in order to identify microRNA’s of interest for therapeutic development,” said Dr. Linda Marbán, Ph.D., CEO of Capricor. “This data was the foundation of our new platform, which has allowed us to expand our reach to engineering exosomes that contain RNAs for targeted therapeutic delivery. Further, this elucidation of the mechanisms that are driving potential potency of our cell therapy, CAP-1002 and the exosomes they secrete, correlate to some of the promising clinical data we have seen to date in Duchenne muscular dystrophy.”
Key findings include:
- Manipulation of cell culture conditions with GSK3 inhibitors leads to upregulation of beta-catenin and downregulation of CD90, leading to a more consistent and potent cell line
- Activation of canonical Wnt signaling correlated with increased enrichment of therapeutically relevant miRs including miR-22 and miR-146a both implicated in cell potency
- This data links the potency of a cell therapy to the contents of the exosomes especially miR-22 and miR-146a
Dr. Marbán continued, “We know that the exosome is nature’s delivery system and can serve to carry a variety of biologic signals directly to the cell. Now, we have demonstrated that cells can make exosomes loaded with specific and powerful biologic signals. From these early studies, we have launched our platform which will include vaccines, treatment of monogenic diseases, as well as other targets. We plan to announce further pipeline expansion of our exosome-based product candidates by mid-2021.”
Source – Globe Newswire