Cyclodextrin for the treatment of lysosomal storage diseases

Patent metadata

Field	Value
Publication number	US-11020422-B2
Application number	US-201715620753-A
Country	US
Kind code	B2
Filing date	Jun 12, 2017
Priority date	Aug 3, 2012
Publication date	Jun 1, 2021
Grant date	Jun 1, 2021

Abstract

The invention provides for methods of treating lysosomal storage disorders and/or reduction of non-cholesterol lipids, using cyclodextrin compounds, including in combination with other therapeutics, including vitamin E.

First claim

What is claimed is:

1. A method of treating Battens disease, Farber disease, mucolipidosis type III (MLIII), mucolipidosis type IV (MLIV), mucopolysaccharidosis type I (MPSI), or mucopolysaccharidosis type VI (MPSVI) in a human, comprising administering to the human in need thereof an effective amount of a hydroxypropyl-β-cyclodextrin compound, or a pharmaceutically acceptable salt, solvate or hydrate thereof, wherein the hydroxypropyl-β-cyclodextrin compound is administered intracranially.

2. The method of claim 1 , wherein the hydroxypropyl-β-cyclodextrin compound comprises from one to ten hydroxypropyl groups.

3. The method of claim 1 , wherein the hydroxypropyl-β-cyclodextrin compound is administered intrathecally, intraventricularly, intracerebrally, or epidurally.

4. The method of claim 3 , wherein the hydroxypropyl-β-cyclodextrin compound is administered intrathecally.

5. The method of claim 1 , wherein the hydroxypropyl-β-cyclodextrin compound is administered in combination with a pharmaceutically acceptable carrier or excipient.

6. The method of claim 1 , wherein the hydroxypropyl-β-cyclodextrin compound comprises an average of four hydroxypropyl groups.

Assignees

• Us Health

Inventors

• Mckew John
• Zheng Wei
• Xu Miao
• Swaroop Manju
• Marugan Juan J

Classifications

• A61K45/06 Primary

  Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca · CPC title

• A61P25/00

  Drugs for disorders of the nervous system · CPC title

• A61P3/08

  for glucose homeostasis (pancreatic hormones A61P5/48) · CPC title

• A61K31/355

  Tocopherols, e.g. vitamin E · CPC title

• C08B37/0012

  Cyclodextrin [CD], e.g. cycle with 6 units (alpha), with 7 units (beta) and with 8 units (gamma), large-ring cyclodextrin or cycloamylose with 9 units or more; Derivatives thereof · CPC title