Small molecule transthyretin ligands for the treatment of dry Age-related Macular Degeneration and orphan Stargardt Disease
Age-related Macular Degeneration (AMD) is the leading cause of blindness in developing countries. AMD occurs in both dry (atrophic) and wet (exudative) forms, with dry AMD accounting for the majority of all AMD cases. However, there are currently no FDA-approved therapies for this disease. Dry AMD is caused by degeneration of photoreceptor cells in the central part of the retina, due to the buildup of cytotoxic lipofuscin bisretinoids, such as A2E, in the eye. This technology describes several small molecule inhibitors that disrupt the formation of A2E. These compounds could be used to treat dry AMD, as well as other degenerative eye diseases related to the buildup of lipofuscin bisretinoids, including orphan Stargardt disease and Best disease.
Describes a library of transthyretin-specific compounds and their mechanism of action that decrease the amount of toxic lipofuscin buildup.Some molecules are already FDA approved for other indicationsPatent Information:Patent Pending (US 20150057320)Tech Ventures Reference: IR CU12294
Therapeutic for ocular diseases caused by lipofuscin build-upTherapeutic for dry age-related macular degenerationTherapeutic for Stargardt diseaseTherapeutic for Stargardt-like macular dystrophyTherapeutic for Best diseaseResearch tool for characterization of the vision cycle
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美国
