IP Library › Granted Patent US 12,268,749
Granted Patent B2
US 12,268,749 · App. 17/266,939 · Granted Apr 8, 2025

Cell-penetrating peptides

Inventors: Matthew Wood (Oxford, GB); Raquel Manzano (Zargoza, ES); Caroline Godfrey (Oxford, GB); Graham McClorey (Oxford, GB); Richard Raz (London, GB); Michael Gait (Cambridge, GB); Andrey Arzumanov (Cambridge, GB); Liz O'Donovan (Ballyanly, IE); Gareth Hazell (Oxford, GB); Ashling Holland (Oxford, GB); Miguel Varela (Oxford, GB)
Assignees: Oxford University Innovation Limited; United Kingdom Research and Innovation
A61K47/645C07K7/08C07K14/00C12N15/113C07K2319/10C12N2310/11C12N2310/3513C12N2320/32
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Quick Facts
Patent No.
US 12,268,749
App. No.
17/266,939
Granted
Apr 8, 2025
Kind
B2
Abstract

The present invention relates to peptides, in particular cell-penetrating peptides, and to conjugates of such cell-penetrating peptides with a therapeutic molecule. The present invention further relates to use of such peptides or conjugates in methods of treatment or as a medicament, especially in the treatment of genetic disorders and in particular muscular dystrophies such as Duchenne muscular dystrophy.

Claims (59)

1. A cell-penetrating peptide consisting of the amino acid sequence

(SEQ ID NO: 27)

RBRRBRRFQILYRBRBR,

(SEQ ID NO: 31)

RBRRBRRYQFLIRBRBR,

(SEQ ID NO: 32)

RBRRBRRILFQYRBRBR,

(SEQ ID NO: 35)

RBRRBRFQILYBRBR,

(SEQ ID NO: 37)

RBRRBRRFQILYRBHBH,

(SEQ ID NO: 38)

RBRRBRRFQILYHBHBR,

(SEQ ID NO: 44)

RBRRBRFQILYRBHBH,

(SEQ ID NO: 29)

RBRRBRFQILYRRBRBR,

(SEQ ID NO: 30)

RBRBRFQILYRBRRBRR,

(SEQ ID NO: 33)

RBRRBRFQILYRBRBR,

(SEQ ID NO: 36)

RBRRBFQILYRBRBR,

(SEQ ID NO: 39)

RBRRBRRFQILYHBRBH,

(SEQ ID NO: 40)

RBRRBRRYQFLIRBHBH,

(SEQ ID NO: 41)

RBRRBRRILFQYRBHBH,

(SEQ ID NO: 42)

RBRHBHRFQILYRBRBR,

(SEQ ID NO: 52)

RBRRBRRFQILYHBHBH,

(SEQ ID NO: 104)

RBRRBRWWWBRBR,or

(SEQ ID NO: 105)

RBRRBRWWPWWBRBR,

wherein B in the amino acid sequences is beta-alanine, and wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsullfonylated, or methylsulfonylated.

2. The cell-penetrating peptide of claim 1 , wherein the N-terminus of the cell-penetrating peptide is acetylated.

3. The cell-penetrating peptide of claim 1 , wherein cell-penetrating peptide consists of the amino acid sequence RBRRBRRFQILYRBRBR (SEQ ID NO: 27), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

4. The cell-penetrating peptide of claim 1 , wherein cell-penetrating peptide consists of the amino acid sequence RBRRBRRYQFLIRBRBR (SEQ ID NO: 31), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

5. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRILFQYRBRBR (SEQ ID NO: 32), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

6. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRFQILYBRBR (SEQ ID NO:35), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

7. The cell-penetrating peptide of claim 6 , wherein the N-terminus of the cell-penetrating peptide is acetylated.

8. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRFQILYRBHBH (SEQ ID NO: 37), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

9. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRFQILYHBHBR (SEQ ID NO: 38), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

10. The cell-penetrating peptide of claim 1 , wherein of the cell-penetrating peptide consists of the amino acid sequence RBRRBRFQILYRBHBH (SEQ ID NO: 44), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

11. The cell-penetrating peptide of claim 10 , wherein the N-terminus of the cell-penetrating peptide is acetylated.

12. The cell-penetrating peptide of claim 1 , wherein of the cell-penetrating peptide consists of the amino acid sequence RBRRBRFQILYRRBRBR (SEQ ID NO: 29, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

13. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRBRFQILYRBRRBRR (SEQ ID NO: 30, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

14. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRFQILYRBRBR (SEQ ID NO: 33, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

15. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBFQILYRBRBR (SEQ ID NO: 36), wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

16. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRFQILYHBRBH (SEQ ID NO: 39, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

17. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRYQFLIRBHBH (SEQ ID NO: 40, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

18. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRILFQYRBHBH (SEQ ID NO: 41, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

19. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRHBHRFQILYRBRBR (SEQ ID NO: 42, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

20. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRRFQILYHBHBH (SEQ ID NO: 52, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

21. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRWWWBRBR (SEQ ID NO: 104, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

22. The cell-penetrating peptide of claim 1 , wherein the cell-penetrating peptide consists of the amino acid sequence RBRRBRWWPWWBRBR (SEQ ID NO: 105, wherein the N-terminus of the cell-penetrating peptide is optionally acetylated, methylated, trifluoroacetylated, trifluoromethylsulfonylated, or methylsulfonylated.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 1, 2024
From: WOOD, MATTHEW; MANZANO, RAQUEL; GODFREY, CAROLINE; MCCLOREY, GRAHAM; RAZ, RICHARD; HAZELL, GARETH; HOLLAND, ASHLING; VARELA, MIGUEL
To: OXFORD UNIVERSITY INNOVATION LIMITED
Reel/Frame 066964/0478 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 1, 2024
From: GAIT, MICHAEL; ARZUMANOV, ANDREY; O'DONOVAN, LIZ
To: UNITED KINGDOM RESEARCH AND INNOVATION
Reel/Frame 066964/0494 →
Priority Claims (1)
GB 1812972 · Aug 9, 2018 · national
Continuity (1)
Related Publication 20210299263A1 · Sep 30, 2021
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