Capricor Therapeutics to present exosome platform updates

Capricor Therapeutics announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore from May 7-11. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency or ARG1-D, a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells. Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro. The data suggests that the Arg1-exosomes were able to not only catalyze arginine efficiently at a lower delivered dose to cells in vitro but also protect the Arg1 protein to be delivered into target cells and retain its enzymatic activity in cells. Taken together, Capricor’s StealthX platform of engineered Arg1-exosomes as tested in vitro have the potential to serve as an enzyme replacement therapy to deliver Arg1 to hepatocytes and possibly have potential clinical benefits for the treatment of ARG1-D.