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Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders
Case ID:
TAB-2554
Web Published:
12/6/2022
Description:
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. These molecules by themselves or in combination with Cyclodextrins (CDs) increase intracellular Ca2+ and enhance exocytosis. Also, the treatment with these compounds reduced the pathological changes in the ultrastructure of LSD cells as observed using electron microscopy analysis. The inventors also found that there is a synergy between CDs and the new tocopherol analogues when tested on the NPC cells and cells from six other lysosomal storage diseases including Wolman, Niemann Pick Type A, Farber, TaySachs, MSIIIB and CLN2 (Batten) diseases. These new tocopherol analogues are as good or better than natural occurring tocopherols and tocotrienols in reducing cholesterol accumulation in several LSDs.
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Direct Link:
https://nih.technologypublisher.com/tech/Novel_Tocopherol_and_Tocopheryl_Quin one_Derivatives_as_Therapeutics_for_Lysosomal_Storage_Disorders
Category(s):
Collaboration
Vaccines
Therapeutics
Reproductive Health
Diagnostics
Research Materials
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For Information, Contact:
Suryanarayana Vepa
Technology Licensing Specialist
NIH Technology Transfer
301-827-7181
sury.vepa@nih.gov
Inventors:
Jingbo Xiao
Wei Zheng
John McKew
Juan Marugan
Keywords:
ANALOGUES
Correctors
DISORDERS
IB6XXX
IBXXXX
IXXXXX
Lysosomal
Storage
Tocopherol
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