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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

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*Abstract

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*Inquiry

Ron KatzColumbia Technology VenturesTel: (212) 854-8444Email: TechTransfer@columbia.edu

*IR

cu14141

*Principal Investigator

*Publications

Petrukhin K. “New therapeutic targets in atrophic age-related macular degeneration.” Expert Opin Ther Targets. 2007 May;11(5):625-39.Dobri N, Qin Q, Kong J, Yamamoto K, Liu Z, Moiseyev G, Ma JX, Allikmets R, Sparrow JR, Petrukhin K. “A1120, a nonretinoid RBP4 antagonist, inhibits formation of cytotoxic bisretinoids in the animal model of enhanced retinal lipofuscinogenesis.” Invest Ophthalmol Vis Sci. 2013 Jan 7;54(1):85-95.Petrukhin K. “Pharmacological inhibition of lipofuscin accumulation in the retina as a therapeutic strategy for dry AMD treatment.” Drug Discov Today Ther Strateg. 2013;10(1):e11-e20.Cioffi CL, Dobri N, Freeman EE, Conlon MP, Chen P, Stafford DG, Schwarz DM, Golden KC, Zhu L, Kitchen DB, Barnes KD, Racz B, Qin Q, Michelotti E, Cywin CL, Martin WH, Pearson PG, Johnson G, Petrukhin K. “Design, synthesis, and evaluation of nonretinoid retinol binding protein 4 antagonists for the potential treatment of atrophic age-related macular degeneration and Stargardt disease.” J Med Chem. 2014 Sep 25;57(18):7731-57.Cioffi CL, Racz B, Freeman EE, Conlon MP, Chen P, Stafford DG, Schwarz DM, Zhu L, Kitchen DB, Barnes KD, Dobri N, Michelotti E2, Cywin CL, Martin WH, Pearson PG, Johnson G, Petrukhin K. “Bicyclic [3.3.0]-Octahydrocyclopenta[c]pyrrolo Antagonists of Retinol Binding Protein 4: Potential Treatment of Atrophic Age-Related Macular Degeneration and Stargardt Disease.”

Country/Region

USA