Capricor Therapeutics, Inc.

Capricor Therapeutics, Inc. is a biotechnology company focused on developing and potentially selling cell and exosome-based treatments. The company primarily targets Duchenne muscular dystrophy ("DMD"), a rare genetic disorder causing progressive muscle degeneration and early death, along with other diseases where current treatments are insufficient. Since its start, the company has invested heavily in its main product candidate, Deramiocel, a cell therapy designed to address heart and skeletal muscle issues related to DMD. They have also advanced their exosome-based technology, built manufacturing capabilities, and supported research and development. The U.S. Food and Drug Administration ("FDA") is currently reviewing Capricor Therapeutics' Biologics License Application ("BLA") for Deramiocel to treat DMD. The FDA's target date for action, under the Prescription Drug User Fee Act ("PDUFA"), is August 22, 2026, for potential approval in the United States. The company currently has no products approved for commercial sale. If approved, Capricor Therapeutics plans to sell Deramiocel in the United States and may partner with other companies for sales in specific international markets. Deramiocel's development for DMD has involved multiple clinical studies, and the company continues activities to support regulatory review, potential U.S. approval, and commercialization preparations. Their core program centers on Deramiocel, a cell therapy made from cardiosphere-derived cells ("CDCs"). These CDCs are a type of cardiac stromal cell obtained from donated human hearts and are intended to treat Duchenne muscular dystrophy. Deramiocel aims to slow disease progression through the CDCs' ability to modulate the immune system, reduce inflammation, promote blood vessel formation, and prevent fibrosis. These effects are partly due to exosomes released by CDCs, which contain active molecules like microRNAs and other signaling factors that can affect gene expression and cellular processes involved in inflammation, fibrosis, and tissue repair.