Capricor Therapeutics, Inc. News: Capricor Therapeutics Announces Intent to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy

About Us

Who We Are

Capricor Therapeutics is a publicly traded (NASDAQ: CAPR) biotechnology company with a mission to develop groundbreaking therapies that make a meaningful impact on patients’ lives. Capricor is focused on the development of biologics, primarily cell and exosome-based technologies to treat or prevent a broad range of diseases. Our lead product, deramiocel (also referred to as CAP-1002) is a cell therapy designed to treat Duchenne muscular dystrophy (DMD), the most severe form of muscular dystrophy, which results in muscle degeneration and premature death. Capricor’s innovative exosome technology is focusing on engineering exosomes to treat or prevent diseases with high unmet medical needs. Our translational approach to product development is based on the strong research foundation provided by our academic partnerships with leading scientists at top-tier research institutions.

Our Portfolio

Capricor’s lead product candidate, deramiocel (CAP-1002), is an allogeneic cell therapy that is currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy.

Deramiocel is comprised of allogeneic cardiosphere-derived cells, or CDCs. The ability of deramiocel to slow disease progression in Duchenne muscular dystrophy lies in the immunomodulatory, anti-inflammatory, pro-angiogenic, 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 (and in a variety of other inflammatory diseases).

Capricor has also established itself as one of the leading companies advancing exosome-based technology. Emerging as an exciting class of potential therapeutic agents and drug delivery vehicles, exosomes are nanosized particles with biologically active contents that are secreted by cells and serve to direct or re-direct the activities of other cells. Capricor is focused on developing a precision-engineered exosome platform technology with the ability to deliver defined sets of effector molecules which exert their effects through defined mechanisms of action. Our StealthX™ platform builds on advances in fundamental RNA and protein science, targeting technology and manufacturing, providing us the opportunity to potentially build a broad pipeline of new therapeutic candidates.

View Our Pipeline