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DC6001 is an investigational small molecule RBP4 (retinol-binding protein 4) antagonist developed by Heronova Pharmaceuticals for the treatment of Stargardt disease. It is designed to disrupt the transport of retinol from the blood to the retina, thereby limiting the accumulation of toxic vitamin A byproducts (bisretinoids) such as lipofuscin, which are hallmarks of Stargardt disease and contribute to retinal degeneration. DC6001 is administered as an oral tablet and is currently undergoing Phase 1 clinical evaluation in healthy subjects to assess safety, pharmacokinetics, and its ability to lower circulating RBP4 levels.
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