Our Science

Expanding the Transformative Potential of CDCs (CAP-1002)

Our core therapeutic technology, CAP-1002, is comprised of cardiosphere-derived cells, or CDCs, which is an endogenous population of stromal cells derived from cells of healthy human hearts. This technology was first discovered and identified in the academic laboratory of Capricor’s scientific founder, Dr. Eduardo Marbán, while he was Chief of Cardiology at The Johns Hopkins University. Since their initial publication in 2007, CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials. The ability of CAP-1002 to slow disease progression in DMD lies in the immunomodulatory, anti-inflammatory, and anti-fibrotic actions of CDCs, which are mediated by secreted exosomes containing bioactive cargo. Among the cargo elements known to be bioactive in CDC exosomes are microRNAs. Collectively, these non-coding RNA species alter gene expression in macrophages and other target cells, dialing down generalized inflammation and stimulating tissue regeneration in DMD.

Ongoing research at Capricor spans the areas of product development and characterization of potential expanded use of CAP-1002. CAP-1002 may be able to work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.