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Over 2 million people in the Untitled States have lost their vision due to vitamin A deposits (1-6).
ALK-001 is a better vitamin A that doesn't form deposits in the eye, which may prevent millions of people from going blind (7-12)
ALK-001 is being developed by Alkeus Pharmaceuticals
1. Zhang D, Mihai DM, Washington I. Vitamin A cycle byproducts explain retinal damage and molecular changes thought to initiate retinal degeneration. Biol Open. 2021;10(11). PMID: 34842275
2. Zhang D, Robinson K, Saad L, Washington I. Vitamin A cycle byproducts impede dark adaptation. J Biol Chem. 2021;297(3):101074. Epub 2021/08/16. doi: 10.1016/j.jbc.2021.101074. PubMed PMID: 34391781; PMCID: PMC8427233.
3. Sears AE, Bernstein PS, Cideciyan AV, Hoyng C, Charbel Issa P, Palczewski K, Rosenfeld PJ, Sadda S, Schraermeyer U, Sparrow JR, Washington I, Scholl HPN. Towards Treatment of Stargardt Disease: Workshop Organized and Sponsored by the Foundation Fighting Blindness. Transl Vis Sci Technol. 2017;6(5):6. PMID: 28920007
4. Washington I, Saad L. The Rate of Vitamin A Dimerization in Lipofuscinogenesis, Fundus Autofluorescence, Retinal Senescence and Degeneration. Adv Exp Med Biol. 2016;854:347-53. PMID: 26427431.
5. Penn J, Mihai DM, Washington I. Morphological and physiological retinal degeneration induced by intravenous delivery of vitamin A dimers in rabbits. Dis Model Mech. 2015;8(2):131-8. PMID: 25504631
6. Mihai DM, Washington I. Vitamin A dimers trigger the protracted death of retinal pigment epithelium cells. Cell Death Dis. 2014;5(7):e1348. PMID: 25058422
7. Zhang D, Robinson K, Washington I. C20D3-Vitamin A Prevents Retinal Pigment Epithelium Atrophic Changes in a Mouse Model. Transl Vis Sci Technol. 2021;10(14):8. PMID: 34878528
8. Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. PMID: 26427432
9 .Charbel Issa P, Barnard AR, Herrmann P, Washington I, MacLaren RE. Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization. Proc Natl Acad Sci U S A. 2015;112(27):8415-20. PMID: 26106163
10. Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D(3)-vitamin A in a swine model. Mol Vis. 2013;19:1677-83. PMID: 23914132
11. Ma L, Kaufman Y, Zhang J, Washington I. C20-D3-vitamin A slows lipofuscin accumulation and electrophysiological retinal degeneration in a mouse model of Stargardt disease. J Biol Chem. 2011;286(10):7966-74. PMID: 21156790
12. Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011;286(10):7958-65. PMID: 21075840