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Invention E-148-2012/0

Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders

Lead Inventor: Wei Zheng (NCATS)
Inventors: Juan Marugan (NCATS), John McKew (NCATS), Jingbo Xiao (NCATS)
Ref. No.: E-148-2012/0

Abstract: Novel tocopherol derivatives and tocopheryl quinone derivatives useful in the decrease of lysosomal substrate accumulation, the restoration of normal lysosomal size, and the treatment of lysosomal storage disorders (LSDs) are provided. The inventors have discovered that tocopherol and tocopheryl quinone derivatives with side chain modifications (such as terminal tri-halogenated methyl groups) exhibit improved pharmacokinetics, modulation of mitochondrial potential and restoration of some LSDs phenotypes.