Peptide, Complex of Peptide and siRNA, and Methods of Use Thereof

Patent metadata

Field	Value
Publication number	US-2018355357-A1
Application number	US-201816110713-A
Country	US
Kind code	A1
Filing date	Aug 23, 2018
Priority date	Dec 18, 2013
Publication date	Dec 13, 2018
Grant date	—

Abstract

A series of peptides and a peptide-siRNA complex are disclosed, wherein the peptide based complex effectively enhances delivery of siRNA molecules into the cells and release of siRNA in the cell, and improves siRNA mediated gene silencing efficiency of cellular targets. Pharmaceutical compositions that include the complex, as well as a use of the complex in the gene therapy field, are also disclosed.

First claim

What is claimed is:

1 . A peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5.

2 . A complex comprising:
a cargo molecule; and a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5.

3 . The complex according to claim 2 , wherein the cargo molecule and the peptide are coupled non-covalently.

4 . The complex according to claim 2 , wherein the particle size of the complex is in a range of from about 20 nm to about 999 nm.

5 . The complex according to claim 2 , wherein the cargo molecule is a nucleic acid.

6 . The complex according to claim 5 , wherein the nucleic acid is a short interfering RNA (siRNA).

7 . The complex according to claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio within a range of from about 1:1 to about 80:1.

8 . The complex according to claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 20:1.

9 . The complex according to claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 40:1.

10 . The complex according to claim 6 , wherein the siRNA is complexed with the peptide at a molar ratio of about 60:1.

11 . A pharmaceutical composition containing a complex comprising:
a cargo molecule; and a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5.

12 . A kit used to deliver drugs to a patient, wherein the kit comprises:
a pharmaceutical composition comprising a complex comprising:
a cargo molecule; and
a peptide comprising a hydrophobic part and a hydrophilic part, wherein the hydrophilic part comprises a positively charged amino acid residue, wherein the peptide is CH 3 (CH 2 ) 16 CONH—B—CONH 2 , and wherein B is SEQ ID NO:4 or 5;
one or more electrolytes; one or more buffers; a delivery device; and a container adapted to mix one or more other agents together.

13 . The kit according to claim 12 , wherein the cargo molecule and the peptide are coupled non-covalently.

14 . The kit according to claim 12 , wherein the particle size of the complex is in a range of from about 20 nm to about 999 nm.

15 . The kit according to claim 12 , wherein the cargo molecule is a nucleic acid.

16 . The kit according to claim 15 , wherein the nucleic acid is a short interfering RNA (siRNA).

17 . The kit according to claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio within a range of from about 1:1 to about 80:1.

18 . The kit according to claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio in a range of from about 20:1 to about 60:1.

19 . The kit according to claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio of about 20:1 or about 40:1.

20 . The kit according to claim 16 , wherein the siRNA is complexed with the peptide at a molar ratio of about 60:1.

Assignees

• Chen Pu
• Positec Power Tools Suzhou Co Ltd

Inventors

• Chen Pu
• Chu Dafeng
• Chen Baoling
• Xu Wen
• Pan Ran

Classifications

• A61P35/00

  Antineoplastic agents · CPC title

• C12N2320/32

  Special delivery means, e.g. tissue-specific · CPC title

• C07K14/47

  from mammals · CPC title

• C12N15/87

  Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation · CPC title

• A61K31/713

  Double-stranded nucleic acids or oligonucleotides · CPC title