# US 2014/0155604 A1 · Peptide nucleic acid derivatives with good cell penetration and strong affinity for nucleic acid

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- Number: US 2014/0155604 A1
- Document ID: US20140155604A1
- Status: Published application
- Kind: A1
- Application number: 14/109,327
- Filed: 2013-12-17
- Published: 2014-06-05
- Earliest claimed: 2008-03-14
- Assignees: Cti Bio
- Applicants: Cti Bio
- Inventors: Shin CHUNG; Heui-Yeon KIM; Hyun-Jin PARK; Mi-Ran KIM; Jong-Ook LEE
- Web page: https://patentgrep.com/patent/US20140155604A1

## CPC

- C07D 473/18: one oxygen and one nitrogen atom, e.g. guanine
- A61P 31/04: Antibacterial agents
- A61P 31/12: Antivirals
- A61P 35/00: Antineoplastic agents
- C07D 473/16: two nitrogen atoms
- C07D 487/04: Ortho-condensed systems
- C07K 14/003: {Peptide-nucleic acids (PNAs)}
- C07D: HETEROCYCLIC COMPOUNDS (macromolecular compounds C08)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)

## Abstract

The present invention provides a novel class of peptide nucleic acid derivatives, which show good cell penetration and strong binding affinity for nucleic acid.

## Claims

### Claim at position 1

1-16. (canceled)

### Claim 17

A compound of Formula VI:
wherein,
R7 is hydrido, N-succinyl, or substituted or non-substituted alkyl radical;
P1 is selected from hydrido, t-butoxycarbonyl, (9H-fluoren-9-yl)methoxy-carbonyl, substituted or non-substituted benzyloxycarbonyl, and substituted or non-substituted arylsulfonyl radical;
P2 is selected from hydrido, t-butoxycarbonyl, (9H-fluoren-9-yl)methoxy-carbonyl, substituted or non-substituted benzyloxycarbonyl, substituted alkyloxycarbonyl, substituted or non-substituted alkyl, amidinyl, 1,3-bis(t-butoxy-carbonyl)amidinyl, 1,3-bis-(benzyloxycarbonyl)amidinyl radical; and,
L1 is a linker represented by Formula V:
wherein,
Q1 and Qm are substituted or non-substituted methylene radical, and Qm is directly linked to the amino radical;
Q2, Q3, . . . , and Qm-1 are independently selected from substituted or non-substituted methylene, oxygen, sulfur, and substituted or non-substituted amino radical; and,
m is an integer from 2 to 15.

### Claim 18

A compound of Formula IX:
wherein,
R10 is hydroxy, substituted or non-substituted alkyloxy, or substituted or non-substituted amino radical;
P2 is selected from hydrido, t-butoxycarbonyl, (9H-fluoren-9-yl)methoxy-carbonyl, substituted or non-substituted benzyloxycarbonyl, substituted alkyloxycarbonyl, substituted or non-substituted alkyl, amidinyl, 1,3-bis(t-butoxy-carbonyl)amidinyl, 1,3-bis-(benzyloxycarbonyl)amidinyl radical; and,
L1 is a linker represented by Formula V:
wherein,
Q1 and Qm are substituted or non-substituted methylene radical, and Qm is directly linked to the amino radical;
Q2, Q3, . . . , and Qm-1 are independently selected from substituted or non-substituted methylene, oxygen, sulfur, and substituted or non-substituted amino radical; and,
m is an integer from 2 to 15.