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CLT-003 is a novel small molecule inhibitor of hypoxia-inducible factor 1 (HIF-1), developed primarily for the treatment of retinal vascular leakage associated with diabetic macular edema (DME) and age-related macular degeneration (AMD). It acts by inhibiting HIF-1 and nuclear factor kappa B (NFκB), which are master transcription factors regulating genes involved in angiogenesis and inflammation, such as VEGF and ICAM-1. Preclinical studies have shown that CLT-003 potently inhibits endothelial cell growth, migration, tube formation, and angiogenesis while reducing retinal vascular leakage in animal models of diabetic retinopathy and AMD. The drug demonstrates anti-inflammatory, anti-angiogenic, and anti-permeability activities with a favorable safety profile. A nanoparticle formulation using PLGA enables sustained ocular delivery after intravitreal injection[1][2][3][4].
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