TCT-370

TCT-370 is an investigational, orally administered small interfering RNA (siRNA) therapeutic being developed by Targeted Cell Therapies (TCT Group) for the treatment of Gaucher disease and other lysosomal storage disorders. The drug candidate utilizes a proprietary delivery platform consisting of yeast-derived glucan particles (GPs), which are hollow, porous microspheres derived from the cell walls of *Saccharomyces cerevisiae*. These particles encapsulate the siRNA payload, protecting it from the harsh environment of the gastrointestinal tract and facilitating targeted delivery to macrophages via Dectin-1 receptor-mediated phagocytosis in the gut-associated lymphoid tissue (GALT). Once internalized by macrophages—the primary cells affected in Gaucher disease—the siRNA is released to silence specific mRNA targets, potentially offering a non-invasive oral alternative to traditional intravenous enzyme replacement therapies (ERT).