Capricor Therapeutics to Present Exosome Platform Updates at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

-Preclinical findings show exosome-based therapeutic approach for the treatment of arginase-1 deficiency (ARG1-D)-

-Presentation to be featured in an oral session being held on May 9, 2024-

SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today 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 27^th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.

“The findings from this preclinical study have further characterized our StealthX exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We believe our StealthX platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads. We continue to focus our exosome platform in two main areas, the development of therapeutics and vaccines, and our goal is to leverage partnership opportunities to further expand and advance these opportunities.”

Study Overview and Findings:

• In this study, exosomes were engineered to express human Arg1 enzyme inside of the exosomes and were evaluated for their in vitro functionality as an approach for the potential treatment of ARG1-D.
• Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro. In addition, the Arg1-exosomes were capable of delivering the Arg1 protein into 293F and HepG2 cells in a time and dose-dependent manner, contrary to human recombinant Arg1 protein alone at the same or higher dose.
• 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.

Presentation details: