IP Library › Granted Patent US 12661371
Granted Patent B2
US 12661371 · App. 19/350,939 · Granted Jun 23, 2026

Hydroxypropyl beta-cyclodextrin compositions and methods

Inventors: Bernardus Nicolaas Machielse (North Potomac, MD); Allan Darling (North Potomac, MD)
Assignee: Mandos LLC
A61K31/724A61K9/0019A61K9/0085A61K9/08
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Quick Facts
Patent No.
US 12661371
App. No.
19/350,939
Granted
Jun 23, 2026
Kind
B2
Abstract

This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.

Claims (30)

1 . A method of treating Niemann-Pick disease comprising administering to a human subject in need thereof a composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein the mixture comprises unsubstituted beta-cyclodextrin (“DS-0”), wherein the composition is pharmaceutically acceptable for administration to the human subject and the composition comprises less than 5 International Units (IU) of endotoxins per gram.

2 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”).

3 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with two hydroxypropyl groups (“DS-2”).

4 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with three hydroxypropyl groups (“DS-3”).

5 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with four hydroxypropyl groups (“DS-4”).

6 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with five hydroxypropyl groups (“DS-5”).

7 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”).

8 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with seven hydroxypropyl groups (“DS-7”).

9 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with eight hydroxypropyl groups (“DS-8”).

10 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with nine hydroxypropyl groups (“DS-9”).

11 . The method of claim 1 , wherein the composition comprises beta-cyclodextrin substituted with ten hydroxypropyl groups (“DS-10”).

12 . The method of claim 1 , wherein administering comprises intrathecal or intravenous injection.

13 . The method of claim 1 , wherein the composition comprises less than 4 International Units (IU) of endotoxins per gram.

14 . The method of claim 1 , wherein the composition comprises less than 3 International Units (IU) of endotoxins per gram.

15 . The method of claim 1 , wherein the composition comprises less than 1 International Units (IU) of endotoxins per gram.

16 . A method of treating Niemann-Pick disease comprising administering to a human subject in need thereof a composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein the composition comprises beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), wherein the composition is pharmaceutically acceptable for administration to the human subject and the composition comprises less than 5 International Units (IU) of endotoxins per gram.

17 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with two hydroxypropyl groups (“DS-2”).

18 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with three hydroxypropyl group (“DS-3”).

19 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with four hydroxypropyl groups (“DS-4”).

20 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with five hydroxypropyl group (“DS-5”).

21 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”).

22 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with seven hydroxypropyl group (“DS-7”).

23 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with eight hydroxypropyl groups (“DS-8”).

24 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with nine hydroxypropyl group (“DS-9”).

25 . The method of claim 16 , wherein the composition comprises beta-cyclodextrin substituted with ten hydroxypropyl groups (“DS-10”).

26 . The method of claim 16 , wherein the composition comprises less than 4 International Units (IU) of endotoxins per gram.

27 . The method of claim 16 , wherein the composition comprises less than 3 International Units (IU) of endotoxins per gram.

28 . The method of claim 16 , wherein the composition comprises less than 1 International Units (IU) of endotoxins per gram.

29 . The method of claim 16 , wherein administering comprises intracerebroventricular injection.

30 . The method of claim 16 , wherein administering comprises intrathecal or intravenous injection.