Capricor Therapeutics Expands Exosome Technology for DMD

Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD).

Capricor’s lead candidate for DMD is CAP-1002, which is composed of cardiosphere-derived cells (CDCs). These are cardiac progenitor cells, meaning they are able to grow and differentiate into mature heart cells. They have shown regenerative properties and an ability to limit inflammation and scarring.

Results from the Phase 1/2 HOPE clinical trial (NCT02485938) showed that one-time administration of CAP-1002 directly into the heart improved heart muscle function in people with DMD. In addition, it improved arm and hand strength and reduced cardiac scarring. The therapy is being further evaluated in the Phase 2 clinical trial HOPE-2 (NCT03406780), which is testing one-year treatment in boys and men on standard-of-care therapy.

Accumulating evidence has suggested that the therapeutic efficacy of CDCs is due, at least in part, to their release of exosomes. These are tiny bags of cellular contents — such as proteins, lipids and RNA molecules — that play a key role in cell-to-cell communication.

“One of the reasons the exosomes are potentially so useful and transformative is their ability to speak the language of a cell,” Linda Marbán, PhD, president and CEO of Capricor, said in a press release.

Capricor is also developing CAP-2003, comprised of CDC-derived exosomes, as a treatment for DMD. Preclinical data have supported the efficacy of the experimental therapy. Capricor is also developing exosome technologies as treatments in sepsis, graft-versus-host disease (a potential complication from stem cell transplantation), and trauma.

In conjunction with the planned expansion of exosome technology development, the company brought on Stephen Gould, PhD, a professor at Johns Hopkins University and an exosome expert, to serve as executive consultant.

“Exosomes are the body’s natural way of sending complex signals between cells and tissues,” Gould said. “As a result, exosome-based vaccines have the potential to elicit more effective immune reactions against infectious agents and cancers, while exosome-based therapeutics have the potential to stabilize drugs and deliver them to their intended site of action.”

Capricor has the expertise to scale up the production of exosome-based products, and to develop viral vaccines, protein therapies, and treatments for inherited diseases, Gould said.

“We are excited by the commitment of Dr. Gould to help us explore the potential of exosome-based vaccines to help prevent human diseases and exosome-based therapeutics in treating human diseases,” Marbán said. “We look forward to announcing more updates shortly which will further outline some of our near-term goals within our exosomes program.”