IP Library Granted Patent US 10,751,329
Granted Patent B2
US 10,751,329 · App. 16/083,016 · Granted Aug 25, 2020

Treatment of CDKL5 disorders with GSK3β inhibitor tideglusib

Inventors: Elisabetta Ciani (Bologna, IT); Claudia Fuchs (Bologna, IT)
Assignee: Alma Mater Studiorum—Universitádi Bologna
A61K31/433A61P25/00
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Quick Facts
Patent No.
US 10,751,329
App. No.
16/083,016
Granted
Aug 25, 2020
Kind
B2
Abstract

Provided herein are methods of treating a CDKL5 deficiency with tideglusib or a derivative thereof.

Claims (17)

1. A method of treating a CDKL5 deficiency in a subject in need thereof, the method comprising:

administering an amount of tideglusib to the subject in need thereof.

2. The method of claim 1 , wherein the amount is an amount effective to increase the dendritic length of newborn granule cells as compared to an untreated control subject or population.

3. The method of claim 1 , wherein the amount is an amount effective to increase the number of dendrite branches of neuronal cells as compared to an untreated control subject or population.

4. The method of claim 1 , wherein the amount is an amount effective to increase granule cell connectivity as compared to an untreated control subject or population.

5. The method of claim 1 , wherein the amount is an amount effective to reduce at least one symptom of the CDKL5 deficiency.

6. The method of claim 1 , wherein the subject is a juvenile.

7. The method of claim 1 , wherein the amount ranges from about 2 mg/kg bodyweight to about 50 mg/kg bodyweight.

8. The method of claim 1 , wherein the amount is the least amount effective to increase the dendritic length of newborn granule cells as compared to an untreated control subject or population.

9. The method of claim 1 , wherein the amount is the least amount effective to increase the number of dendrite branches of neuronal cells as compared to an untreated control subject or population.

10. The method of claim 1 , wherein the amount is the least amount effective to increase granule cell connectivity as compared to an untreated control subject or population.

11. The method of claim 1 , wherein the amount is the least amount effective to reduce at least one symptom of the CDKL5 deficiency.

12. The method of claim 1 , wherein the least effective amount ranges from about 2 mg/kg bodyweight to 50 about mg/kg bodyweight.

13. The method of claim 1 , wherein the amount is delivered orally, intravenously, subcutaneously, intraventricularly, or intramuscularly.

14. The method of claim 1 , wherein the tideglusib is included in a pharmaceutical formulation.

15. The method of claim 1 , wherein the tideglusib is administered in at least two dosages administered sequentially.

16. The method of claim 1 , wherein the tideglusib is administered every other day.

Assignments (2)
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →