GenSci117

GenSci117 (also known as GenSciP117) is an inhaled small interfering RNA (siRNA) therapeutic being developed by North America Genescience (GeneScience Pharmaceutical) for the treatment of idiopathic pulmonary fibrosis (IPF). Developed using GenSci's extrahepatic siRNA GPODS™ platform, GenSci117 is designed to specifically target and silence matrix metalloproteinase 7 (MMP7) mRNA in lung tissue. MMP7 is a key enzyme involved in epithelial-mesenchymal transition, fibrocyte migration, and fibroblast proliferation, and its elevated levels are highly predictive of disease progression and mortality in IPF patients. By delivering the siRNA directly to the lungs via inhalation, GenSci117 achieves potent, localized knockdown of MMP7 while minimizing systemic exposure and off-target toxicity in extra-pulmonary organs like the kidneys. The program is currently in IND-enabling development.