LEM-S401

LEM-S401 is an investigational siRNA gene therapy designed to treat and prevent fibrotic skin diseases, particularly hypertrophic scars and keloids. It utilizes a proprietary nanoparticle-based delivery system called DegradaBALL, developed by Lemonex, to encapsulate and deliver small interfering RNA (siRNA) targeting connective tissue growth factor (CTGF) directly to fibroblasts. By silencing CTGF expression, which drives abnormal collagen production in fibrotic conditions, LEM-S401 aims to inhibit excessive extracellular matrix deposition and scar formation. Preclinical studies demonstrated durable knockdown of CTGF and reduced collagen types I and III in models of skin fibrosis. In a first-in-human Phase 1 trial, subcutaneous administration of LEM-S401 was well tolerated with only mild local reactions; no significant systemic exposure or adverse events were observed[1][2][5][8]. The drug is being developed for the treatment of hypertrophic scars but may have broader applications across other fibrotic diseases.