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Small molecule therapeutics for dry Age-related Macular Degeneration and related ocular diseases


Summary

Dry (atrophic) Age-related Macular Degeneration (AMD) is a leading cause of blindness and is characterized by the buildup of lipofuscin that induces degeneration of the retinal pigment epithelium (RPE). Despite years of research and clinical trials, no therapies have been developed for the treatment of AMD and related diseases, such as Stargardt disease (STGD). This technology describes a small molecule (BPN-14967) that inhibits lipofuscin formation in the retina through modulation of the visual cycle retinoids. This technology provides an efficient means to treat AMD and other related ocular diseases, many of which have very limited treatment options. Furthermore, this technology includes a well-differentiated screening strategy that has yielded additional, promising drug candidates for common forms of macular degeneration. As such, this technology has the potential to produce many effective treatments that will have significant societal benefit while filling an important void in the medical field.


Technology Benefits

Improved potency and pharmacokinetic profileReduced risk of side effectsSimple drug delivery; orally availableApplicable to multiple forms of macular degenerationPatent Information:Patents: (8,980,924) (US 20140031392)(PCT/US2014/026813)(PCT/US2014/026523)(PCT/US14/26818(PCT/US14/26699)(PCT/US2014/026730) Tech Ventures Reference: 2817, CU12295, CU13230, CU13231, CU13232, CU13233, CU13234, CU14141


Technology Application

Therapeutic for diseases caused by lipofuscin build-upTherapeutic for dry age-related macular degeneration (AMD) and Stargardt disease Potential therapeutic for Best disease and other forms of macular degenerationResearch tool for development of other RBP4 agonists and characterization of the RBP4 binding site


Detailed Technology Description

None


Country/Region

USA

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