# US 10,370,348 B2 · Tocopherol and tocopheryl quinone derivatives as correctors of lysosomal storage disorders

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- Number: US 10,370,348 B2
- Document ID: US10370348B2
- Status: Granted
- Kind: B2
- Application number: 15/608,753
- Filed: 2017-05-30
- Published: 2018-04-19
- Granted: 2019-08-06
- Earliest claimed: 2012-11-16
- Assignees: The United States of America, as represented by the Secretary, Department of Health and Human Services
- Applicants: The United States of America, as represented by the Secretary, Dept. of Health and Human Services
- Inventors: Juan Jose Marugan; Wei Zheng; Jingbo Xiao; John McKew
- Web page: https://patentgrep.com/patent/US10370348B2

## CPC

- C07D 311/58: other than with oxygen or sulphur atoms in position 2 or 4
- A61K 31/122: having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
- A61K 31/335: having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K 31/35: having six-membered rings with one oxygen as the only ring hetero atom
- A61K 31/353: 3,4-Dihydrobenzopyrans, e.g. chroman, catechin
- A61K 31/355: Tocopherols, e.g. vitamin E
- A61K 31/724: Cyclodextrins
- A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00)
- A61P 3/06: Antihyperlipidemics
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C07C 50/02: with monocyclic quinoid structure
- C07C 50/06: with unsaturation outside the quinoid structure
- C07C 50/24: containing halogen
- C07D 311/72: 3,4-Dihydro derivatives having in position 2 at least one methyl radical and in position 6 one oxygen atom, e.g. tocopherols
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C07C: ACYCLIC OR CARBOCYCLIC COMPOUNDS

## Abstract

The subject invention relates to improved tocopheryl quinine derivatives and tocopherol derivatives having improved pharmacokinetics in vivo that can, in some embodiments, be useful in the treatment of Lysosomal Storage Disorder, restoration of normal mitochondrial ATP production, modulation of intracellular calcium ion concentration and other treatments or therapies. The tocopheryl quinone derivatives and tocopherol derivatives have side chains that have terminally halogenated carbon atoms.

## Claims

### Claim 1

A tocopheryl quinone derivative of formula (I):
wherein
R1, R2, and R3 independently are hydrogen, halogen, alkyl, or fluoroalkyl;
Ra and Rb independently are hydrogen or absent in each repeat;
X is halogen; and
the dotted line bonds indicate single or double bonds.