IP Library Granted Patent US 10,584,318
Granted Patent B2
US 10,584,318 · App. 15/954,021 · Granted Mar 10, 2020

TATk-CDKL5 fusion proteins, compositions, formulations, and use thereof

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Quick Facts
Patent No.
US 10,584,318
App. No.
15/954,021
Granted
Mar 10, 2020
Kind
B2
Abstract

Disclosed herein are compositions and formulations containing a TATk-CDKL5 fusion protein. Also disclosed are methods of producing a TATk-CDKL5 fusion protein from vectors containing a TATk-CDKL5 cDNA and methods of transducing cells with the vectors containing a TATk-CDKL5 cDNA and the TATk-CDKL5 fusion protein.

Claims (24)

1. A method of treating a CDKL5 deficiency, Rett syndrome or a Rett syndrome variant, the method comprising administering a fusion protein to a subject in need thereof, wherein the fusion protein comprises:

a CDKL5 polypeptide sequence, wherein the CDKL5 polypeptide sequence comprises SEQ ID NO: 2; and

a TATκ polypeptide sequence, wherein the TATκ polypeptide sequence comprises SEQ ID NO: 4, wherein the TATκ polypeptide is operatively coupled to the CDKL5 polypeptide.

2. The method of claim 1 , wherein the fusion protein further comprises an Igk-chain leader sequence polypeptide, wherein the Igk-chain leader sequence is operatively coupled to the CDKL5 polypeptide.

3. The method of claim 1 , wherein the fusion protein further comprises a reporter protein polypeptide, wherein the reporter protein polypeptide is operatively coupled to the CDKL5 polypeptide.

4. The method of claim 1 , wherein the fusion protein further comprises a protein tag polypeptide, wherein the protein tag polypeptide is operatively coupled to the CDKL5 polypeptide.

5. The method of claim 1 , wherein the fusion protein has a polypeptide sequence comprising SEQ ID NO: 8 or SEQ ID NO: 10.

6. The method of claim 1 , wherein administering the fusion protein increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

7. The method of claim 1 , wherein administering the fusion protein reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

8. The method of claim 1 , wherein the subject has a CDKL5 deficiency.

9. The method of claim 1 , wherein the subject has Rett syndrome.

10. The method of claim 1 , wherein the subject has a Rett syndrome variant.

11. A method of treating a CDKL5 deficiency, Rett syndrome or a Rett syndrome variant, the method comprising administering a fusion protein to a subject in need thereof, wherein the fusion protein comprises:

a CDKL5 polypeptide sequence, wherein the CDKL5 polypeptide sequence comprises SEQ ID NO: 16; and

a TATκ polypeptide sequence, wherein the TATκ polypeptide sequence comprises SEQ ID NO: 4, wherein the TATκ polypeptide is operatively coupled to the CDKL5 polypeptide.

12. The method of claim 11 , wherein the fusion protein further comprises an Igk-chain leader sequence polypeptide, wherein the Igk-chain leader sequence is operatively coupled to the CDKL5 polypeptide.

13. The method of claim 11 , wherein the fusion protein further comprises a reporter protein polypeptide, wherein the reporter protein polypeptide is operatively coupled to the CDKL5 polypeptide.

14. The method of claim 11 , wherein the fusion protein further comprises a protein tag polypeptide, wherein the protein tag polypeptide is operatively coupled to the CDKL5 polypeptide.

15. The method of claim 11 , wherein the fusion protein has a polypeptide sequence comprising SEQ ID NO: 12 or SEQ ID NO: 14.

16. The method of claim 11 , wherein administering the fusion protein increases neurite growth, elongation, branch number, or branch density in the brain of a treated subject as compared to an untreated control subject.

17. The method of claim 11 , wherein administering the fusion protein reduces neuron apoptosis in the brain of a treated subject as compared to an untreated control subject.

18. The method of claim 11 , wherein the subject has a CDKL5 deficiency.

19. The method of claim 11 , wherein the subject has Rett syndrome.

20. The method of claim 11 , wherein the subject has a Rett syndrome variant.

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 →