Cell Therapy

Our core therapeutic technology is based on the cardiosphere-derived cell, or CDC, a type of cardiac progenitor cell that composes a minor fraction of the cardiac muscle cell population and was first identified in the academic laboratory of Capricor’s scientific founder, Dr. Eduardo Marbán. Since the initial report in 2007, CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials. CDCs have been shown to exert potent immunomodulatory activity and alters the immune system’s activity to encourage cellular regeneration. 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. Pre-clinical and clinical data support the therapeutic concept of administering CDCs as a means to address conditions in which the heart or skeletal muscle has been damaged.

What are CDCs?

CDCs are derived from cardiospheres, or CSps, which are self-assembling multicellular clusters which contain both primitive cells and committed progenitors for the three major cell types present in the heart. The relatively large size of CSps makes them less suitable than CDCs for intracoronary or intravenous route of administration. 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. It has been shown that CDCs exert several effects that can mitigate damage and promote repair and regeneration at sites of injury, including anti-scarring, anti-programmed cell death and blood vessel growth functions. The cumulative clinical experience with CDCs has provided initial validation of their potential benefits and is characterized by favorable safety and tolerability.

CDC’s Unique Mechanism of Action

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. CAP-1002 stimulates diverse and lasting changes in cellular behavior by using the natural repair mechanisms of our own natural biological processes.

CAP-1002 Attributes

CAP-1002 could be an important tool in the toolbox to treat Duchenne muscular dystrophy. 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.

Manufacturing

The manufacturing process for CAP-1002, Capricor’s allogeneic CDC product candidate, starts with tissue taken from donor hearts. Following expansion, processing, release testing, and quality review, CAP-1002 is cryopreserved (stored at a low temperature), allowing an inventory of “off the shelf” product to be available for administration to many patients.

Capricor has secured exclusive worldwide IP rights to CAP-1002. It also has secured FDA designations for CAP-1002 for the treatment of DMD. These are:

  • Orphan Drug Designation, which gives the company seven-year market exclusivity upon approval of CAP-1002 for DMD.

  • Rare Pediatric Disease Designation, which means that if CAP-1002 is approved first for use in DMD, the company can secure a priority review voucher to fast-track a potential future therapy.

  • Regenerative Medicine Advanced Therapy (RMAT) designation, which makes therapies eligible for the same actions to expedite the development and review of a marketing application that are available to drugs that receive breakthrough therapy designation – including increased meeting opportunities, early interactions to discuss any potential surrogate or intermediate endpoints and the potential to support accelerated approval. The RMAT designation is an expedited program established under the 21st Century Cures Act to foster the development and approval of regenerative medicine products intended for the treatment of serious and life-threatening diseases and conditions.