Capricor Therapeutics to Update on Exosome Platform at ASGCT Annual Meeting

Capricor Therapeutics, a biotech firm at the forefront of cell and exosome-based treatments for rare diseases, has announced a significant milestone. Their abstract, featuring preclinical data on the therapeutic potential of their StealthX™ exosome platform, has been selected for an oral presentation at the ASGCT 27th Annual Meeting in Baltimore, scheduled for May 7-11, 2024. This study unveils a new exosome-based therapeutic approach for arginase-1 deficiency (ARG1-D), a rare genetic metabolic disorder caused by the lack of the enzyme arginase in the liver and red blood cells.

Linda Marbán, Ph.D., CEO of Capricor, emphasized the groundbreaking nature of the findings. She highlighted that the StealthX™ platform could pave the way for innovative enzyme-replacement therapies. This technology aims to address the enzyme deficiencies by using exosomes as delivery vehicles, capable of carrying various therapeutic payloads. Marbán noted the broad applicability of the StealthX™ platform in developing new therapeutics and vaccines. The company is keen on leveraging partnerships to expand and advance its exosome technology.

The study conducted by Capricor involved engineering exosomes to express the human Arg1 enzyme, which was then tested for its functionality in vitro as a potential treatment for ARG1-D. The results were promising: the Arg1-exosomes were enzymatically active and capable of converting arginine into urea in vitro. Moreover, these exosomes successfully delivered the Arg1 protein into 293F and HepG2 cells in a time and dose-dependent manner, outperforming human recombinant Arg1 protein alone, even at similar or higher doses.

The data suggest that Arg1-exosomes not only catalyze arginine efficiently at lower doses but also protect the Arg1 protein, ensuring its delivery and enzymatic activity within target cells. These findings indicate that Capricor's StealthX™ platform, through engineered Arg1-exosomes tested in vitro, holds potential as an enzyme replacement therapy. This could be pivotal in delivering Arg1 to hepatocytes, potentially offering clinical benefits for ARG1-D treatment.

The abstract titled "Exosome-mediated intracellular delivery of arginase-1: a potential application for the treatment of arginase-1 deficiency" will be presented by Li-En Hsieh, Ph.D., Principal Scientist, on May 9, 2024, at 4:45 p.m. ET during the session on Exosomes, Virus-like Particles, and LNPs.

Capricor Therapeutics is a biotechnology company focused on developing transformative cell and exosome-based therapies to revolutionize treatments for rare diseases. Their leading product candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy currently in Phase 3 clinical development for Duchenne muscular dystrophy (DMD). CAP-1002 has shown promising immunomodulatory, antifibrotic, and regenerative effects in preclinical and clinical studies, specifically for dystrophinopathies and heart disease.

Capricor is also advancing its proprietary StealthX™ platform in preclinical development, targeting areas such as vaccinology, and the delivery of oligonucleotides, proteins, and small molecule therapeutics. The ultimate goal is to treat and prevent a broad spectrum of diseases using this cutting-edge exosome technology.

Capricor remains committed to pushing the boundaries of medical science to develop transformative treatments for those in need. Their dedication to innovation and collaboration continues to drive their mission forward.