ND-L02-s0201

ND-L02-s0201 is an investigational oligonucleotide drug formulated as a lipid nanoparticle encapsulating a synthetic, double-stranded small interfering RNA (siRNA) that targets and inhibits the expression of heat shock protein 47 (HSP47), a collagen-specific chaperone. HSP47 plays a key role in collagen synthesis and secretion, processes central to the development of fibrosis in organs such as the liver and lungs. By silencing HSP47 expression, ND-L02-s0201 aims to reduce excessive collagen accumulation associated with fibrotic diseases. The drug uses a retinoid-conjugated targeting agent within its lipid nanoparticle formulation to enhance uptake by hepatic stellate cells or lung myofibroblasts. It has been evaluated in multiple clinical trials for conditions including advanced liver fibrosis (such as non-alcoholic steatohepatitis [NASH] and hepatitis C virus [HCV]-related fibrosis) and idiopathic pulmonary fibrosis (IPF). ND-L02-s0201 was granted FDA Fast Track designation for both NASH and HCV liver fibrosis[1][2][3][4][5][6][7][8].