Houman David Hemmati, MD PHD is the Vice President of Medical and Clinical Development for New Therapies at Capricor Therapeutics, Inc. in Beverly Hills, California. In this fascinating talk, he discussed the clinical trials at Capricor and the potential of these biotherapeutics in the pharmaceutical space. Capricor has three platforms: CDCs, Peptide therapy and microRNA exosomes:
The Heart of the Matter
This is a summary of the trials underway:
How do they do this? In 2012 they showed as part of their caduceus study that CDCs reduced scar size and increased healthy heart muscle. Not surprisingly, smaller scars have better outcomes. But waiting on donor hearts that aren’t transplanted is not the most viable business model. So they moved to researching exosomes.
Exosomes are small vesicles (30–150 nm) with a sophisticated RNA and protein profile. They are secreted by all cell types in culture and are found to occur naturally in body fluids, including blood, saliva, urine, CSF, and breast milk. Nanoparticles are not cells so do not have the same challenges faced by cell therapy (ie: fragility, immunologic memory, etc). They can modulate gene expression, transcription and translation while maintaining homeostasis. They clearly carry a future potential in tissue repair. They are a novel, cell-free biologic delivery vehicle with a natural capacity to protect their cargo and get manufactured relatively simply. Look out, there’s a new biotherapeutic in town – and it could be dirt cheap compared with cell therapies.