CDCs have been shown to exert potent immunomodulatory activity and alters the immune system’s activity to encourage cellular regeneration. These are: CDCs are sufficiently small that, within acceptable dose limits, they can be infused into a coronary artery or into the peripheral vasculature.Since the discovery of the cardiosphere over a decade ago, basic research on CSPs and CDCs has generated a large and growing body of peer-reviewed literature.

We are currently developing allogeneic CDCs (CAP-1002) as a product candidate for the treatment of Duchenne muscular dystrophy and investigating their effects on skeletal and cardiac function. IC has numerous advantages over NOGA®, including 1) no damage to the myocardium, 2) faster treatment, with greater ability to treat acute cases and 3) lower cost of treatment, as there is minimal addition clinician training and no additional capital equipment or software required. From The Heart To The Heart Within cardiac stem cell therapy, Capricor’s cells have several differentiating characteristics that we believe makes them more efficacious, cost effective and easier to deliver than other cell types. This was demonstrated in a 2012 Cedars-Sinai mouse study comparing CDCs head-to-head with other stem cell types (BM-MSCs, AD-MSCs, Low BM-MNCs, High BM-MNCs). While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function in Duchenne muscular dystrophy, there will still be significant inflammation and fibrosis, which can offset the restorative effects.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. At three weeks, CDCs showed the greatest ejection fraction (only group where ejection fraction improvement reached statistical significance), the highest cell engraftment and cardiac differentiation rates, the least degree of adverse remodeling, and the lowest rate of cell apoptsis. This entails electronic mapping of the heart followed by direct injection of the cells into the myocardium, via a catheter. The cumulative clinical experience with CDCs has provided initial validation of their potential benefits and is characterized by favorable safety and tolerability.Based on our understanding of the mechanism of action of CAP-1002 which has been seen in pre-clinical models of DMD, we believe that CAP-1002 has the potential to decrease inflammation and muscle degeneration while exerting positive effects on muscle regeneration, all of which may translate into patients retaining muscle function for a longer period of time. First, as thousands of doses can be created from one donor heart, the therapy can be available “off-the-shelf.” Secondly, there is no need for an invasive biopsy to harvest tissue. Capricor has developed a therapy called CAP-1002 using cells derived from heart stem cells, called cardiospheres. It also has secured FDA designations for CAP-1002 for the treatment of DMD. Within cardiac stem cell therapy, Capricor’s cells have several differentiating characteristics that we believe makes them more efficacious, cost effective and easier to deliver than other cell types.Allogeneic stem cells, which are derived from donor tissue, offer several advantages over autologous alternatives (derived from patient’s own tissue). CAP-1002 stimulates diverse and lasting changes in cellular behavior by using the natural repair mechanisms of our own natural biological processes.CAP-1002 could be an important tool in the toolbox to treat Duchenne muscular dystrophy. The relatively large size of CSps makes them less suitable than CDCs for intracoronary or intravenous route of administration. Finally, the cost of production is significantly less than an autologous product.The majority of cardiac stem cell therapies utilize the NOGA® MyoStar intramyocardial injection catheter system for delivery. Since the initial report in 2007, CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered […] Our core therapeutic technology is based on the cardiosphere-derived cells, or CDC’s, a type of cardiac derived cell therapy that was first identified in the academic laboratory of Capricor’s scientific founder, Dr. Eduardo Marbán.