IP Library › Granted Patent US 12667588
Granted Patent B2
US 12667588 · App. 19/350,933 · Granted Jun 30, 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
View Patent ↗
Loading inventors, assignments & file history…
Monitor This Case
Get email alerts when status or documents change.
Order Certified Copies
Most orders are placed with the USPTO same day — all within 24 business hours.
Order via The Patent Place →
Pre-filled with this patent's details
Quick Facts
Patent No.
US 12667588
App. No.
19/350,933
Granted
Jun 30, 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 (28)

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 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 administering comprises intrathecal or intravenous injection.

3 . The method of claim 1 , wherein administering comprises intracerebroventricular injection.

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

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

6 . The method of claim 1 , wherein the composition comprises less than 2 International Units (IU) of endotoxins per gram.

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

8 . The method of claim 1 , wherein the composition comprises less than 0.5 International Units (IU) of endotoxins per gram.

9 . The method of claim 1 , wherein the composition comprises less than 0.3 International Units (IU) of endotoxins per gram.

10 . The method of claim 1 , wherein the composition comprises less than 0.2 International Units (IU) of endotoxins per gram.

11 . The method of claim 1 , wherein the composition comprises less than 0.1 International Units (IU) of endotoxins per gram.

12 . The method of claim 1 , wherein the composition comprises less than 0.05 International Units (IU) of endotoxins per gram.

13 . The method of claim 1 , wherein the composition comprises less than 1 ppm propylene oxide.

14 . The method of claim 1 , wherein the composition comprises less than 0.8 ppm propylene oxide.

15 . The method of claim 1 , wherein the composition comprises less than 0.6 ppm propylene oxide.

16 . The method of claim 1 , wherein the composition is suitable for lumbar injection.

17 . The method of claim 1 , wherein the composition is suitable for administration directly to the cerebrospinal fluid of infants.

18 . The method of claim 1 , wherein the composition is suitable for administration directly to the cerebrospinal fluid of children.

19 . The method of claim 1 , wherein the composition comprises less than 3% unsubstituted beta-cyclodextrin (“DS-0”).

20 . The method of claim 1 , wherein the composition comprises less than 2% unsubstituted beta-cyclodextrin (“DS-0”).

21 . The method of claim 20 , wherein the composition comprises less than 4% beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”).

22 . The method of claim 1 , wherein the composition comprises less than 1.5% unsubstituted beta-cyclodextrin (“DS-0”).

23 . The method of claim 1 , wherein the composition comprises less than 1% unsubstituted beta-cyclodextrin (“DS-0”).

24 . The method of claim 1 , wherein the composition comprises less than 1% unsubstituted beta-cyclodextrin (“DS-0”) and beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), collectively.

25 . The method of claim 1 , wherein the composition comprises less than 4% beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”).

26 . 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 is pharmaceutically acceptable for injection, the composition comprises less than 5 Endotoxin Units (EU) of endotoxins per gram, no more than 1.0% propylene glycol, and less than 1 ppm of propylene oxide.

27 . The method of claim 26 , wherein the composition comprises less than 2% unsubstituted beta-cyclodextrin (“DS-0”).

28 . The method of claim 26 , wherein the composition comprises less than 4% beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”).