-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
“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: | ||
Title: | Exosome-mediated intracellular delivery of arginase-1: a potential application for the treatment of arginase-1 deficiency | |
Oral session: | Exosomes, Virus like Particles and LNPs | |
Presenting author: | Li-En Hsieh, Ph.D., Principal Scientist | |
Date: | ||
Abstract number: | 223 | |
The abstract will be made available on the publications section of the
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