Amicus Therapeutics preclinical data

AT3375 as monotherapy increased glucocerebrosidase (GBA; GCase) activity in Gaucher's disease patient-derived cells with greater potency compared to AT2101. AT3375 is a next-generation version of the company's AT2101, a small molecule chaperone that binds to GCase. Additionally, AT3375 in combination with enzyme replacement therapy (ERT) increased levels of active GCase in both plasma and Gaucher's disease-relevant tissues (liver, spleen and lung) vs. AT2101 plus ERT. In mice, Amicus said that AT3375 increased brain GCase levels with greater potency than AT2101. Data were presented at the Lysosomal Disease Network World Symposium in San Diego. ...