Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. So what is Deramiocel and what does it do?
What is Deramiocel?
Deramiocel (CAP-1002), developed by Capricor Therapeutics, is a promising treatment under investigation for Duchenne Muscular Dystrophy (DMD), a severe and progressive genetic disorder characterized by muscle degeneration and weakness.
Mechanism of Action
Deramiocel is a cell-based therapy derived from cardiac-derived mesenchymal stem cells (MSC). These stem cells have the ability to secrete factors that support muscle repair and regeneration. The goal of Deramiocel is to harness the regenerative properties of these stem cells to reduce inflammation, promote tissue repair, and potentially regenerate damaged muscle tissue in individuals suffering from DMD.
Key Points of Deramiocel’s Potential Use in DMD:
- Cell-based Therapy: Deramiocel uses stem cells isolated from heart tissue, which are then processed and administered to patients. The mesenchymal stem cells are thought to promote healing by releasing proteins and cytokines that reduce inflammation and stimulate muscle regeneration.
- Targeting Muscle Regeneration: DMD leads to progressive damage and weakness in skeletal muscles. Deramiocel may aid in regenerating muscle tissue by stimulating the body’s natural healing processes and creating an environment that encourages muscle cell survival and growth.
- Potential for Slowing Disease Progression: By addressing inflammation and supporting the repair of muscle tissue, Deramiocel could help slow down the progression of muscle degeneration in DMD, potentially improving mobility and quality of life for patients.
- Delivery Method: The cells are typically administered through an injection, allowing them to reach the damaged muscle tissues and release the reparative factors.
Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy
As of the latest updates, Capricor Therapeutics has been conducting clinical trials to assess the safety and efficacy of Deramiocel for DMD. The results from these trials will determine whether this treatment can become a viable therapeutic option for individuals with Duchenne Muscular Dystrophy.
If proven effective, Deramiocel could become a significant advancement in the treatment of DMD, offering new hope to patients and families affected by this challenging condition.
