exosome based peptide therapeutics / Evox Therap - LARVOL DELTA

[VIRTUAL] Using Novel Intein Technology to Deliver Exosome-Based Therapeutics (ASGCT 2021) - "Here we describe targeted extracellular vesicle (EV) loading of entirely soluble ASL when expressed as a cleavable ASL fusion protein, compared to wild-type ASL which is exclusively intracellular.In addition, we show in vitro confirmation of the ability of complex engineered proteins to fold correctly and retain activity comparable to wild-type human ASL enzyme. Finally, we present data to demonstrate functionality of exosome-delivered soluble ASL protein through in vivo modification of biomarkers of disease in a hypomorphic knock-in AslNeo/Neo murine model.Lastly, we also demonstrated proof of concept of the modularity of this intein-based design through successful loading of protein cargos for arginosuccinate synthetase deficiency (ASSD), a second urea cycle disorder caused by a mutation in the ASS1 gene leading to a deficiency in hepatic enzyme arginosuccinate synthetase (ASS) that results in high levels of citrulline and ammonia."

Metabolic Disorders • Rare Diseases • ASS1