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URL-903 is a small molecule therapeutic currently in preclinical development by United Research Laboratories for the treatment of lymphangioleiomyomatosis (LAM). LAM is a rare, progressive cystic lung disease that primarily affects women, characterized by the proliferation of abnormal smooth muscle-like cells (LAM cells) throughout the lungs and other organs. These cells often harbor mutations in the TSC1 or TSC2 genes, leading to hyperactivation of the mTOR signaling pathway. URL-903 is part of a pipeline focused on addressing the underlying pathophysiology of LAM, aiming to provide a novel treatment option for a condition with limited therapeutic interventions. As of current reports, the specific molecular target of URL-903 has not been publicly disclosed.
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