LAM-001

LAM-001 is a proprietary, investigational, inhaled formulation of sirolimus (also known as rapamycin), developed to deliver the drug directly to the lungs. Sirolimus is an mTOR inhibitor with immunosuppressive and antiproliferative properties. The inhaled formulation aims to minimize systemic exposure and associated toxicities seen with oral sirolimus while maximizing therapeutic effects in pulmonary diseases. LAM-001 modulates both the mTOR and BMPR2 pathways—reducing smooth muscle cell hyperproliferation and ameliorating endothelial cell dysfunction in the pulmonary vasculature—which are key drivers of disease progression in conditions like pulmonary arterial hypertension (PAH) and bronchiolitis obliterans syndrome (BOS). It is currently being evaluated in Phase 2 clinical trials for PAH and BOS[1][3][4][5][6][7][8].

Other names

sirolimus inhaledrapamycin inhaled