Moss-GAA / Greenovation Biotech - LARVOL DELTA

Uptake of moss-derived human recombinant GAA in Gaa mice. (PubMed, JIMD Rep) - "The only available treatment to date is an enzyme replacement therapy (ERT) with alglucosidase alfa, a recombinant human GAA (rhGAA) modified with mannose-6-phosphate (M6P), which is internalized via M6P-mediated endocytosis. We investigated general effects as well as the uptake into different organs following short-term treatment. Our results do confirm that moss-GAA reaches the target disease organs and thus might have the potential to be an alternative or complementary ERT to the existing one."

Journal • Preclinical • Lysosomal Storage Diseases • Metabolic Disorders • Pompe Disease • Rare Diseases