IP Library Granted Patent US 6,900,308
Granted Patent B2
US 6,900,308 · App. 10/195,980 · Granted May 31, 2005

α-modified nucleoside triphosphates

Assignee: ISIS Pharmaceuticals, Inc.
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Quick Facts
Patent No.
US 6,900,308
App. No.
10/195,980
Granted
May 31, 2005
Kind
B2
Abstract

The present invention is directed to α-modified nucleoside triphosphates and methods for their preparation. The present invention also provides for methods of inhibiting RNA and viral replication in cells, as well as methods for treating viral infections.

Claims (54)

1. A compound of formula I

wherein:

Y 1 is S and Y 2 is NHR 1 ; or

Y 1 is Se and Y 2 is SH;

Y 3 is NHR 1 , OH, C 1 -C 12 alkyl, or C 1 -C 12 O-alkyl;

R 1 is hydrogen, substituted or unsubstituted C 1 -C 12 alkyl, substituted or unsubstituted C 2 -C 12 alkenyl, substituted or unsubstituted C 2 -C 12 alkynyl, substituted or unsubstituted C 5 -C 20 aralkyl, substituted or unsubstituted C 5 -C 20 alkylaryl, substituted or unsubstituted C 5 -C 20 heterocycloalkyl, substituted or unsubstituted C 5 -C 20 aryl, substituted or unsubstituted C 5 -C 20 heteroaryl, said substituted moieties comprising one or more substituents selected from hydrogen, halo, C 1 -C 12 alkyl, C 2 -C 12 alkenyl, C 2 -C 12 alkynyl, C 5 -C 20 aryl, C 1 -C 12 alkoxy, alkoxyalkyl, O-alkylaminoalkyl, O-alkylimidazolyl, S-alkyl, amino, alkylamino, dialkylamino, arylamino, diarylamino, C 5 -C 20 aryl, C 5 -C 20 heteroaryl, C 1 -C 12 cycloalkyl, C 1 -C 12 heterocyclolalkyl, O-aryl, S-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, nitro, cyano, aldehyde, carboxylic acid, hydroxy, alkylcarbonyl, aminocarbonyl, or mercapto;

Bx is an optionally protected heterocyclic base moiety;

one of X 1 and X 2 is H, hydroxyl, a protected hydroxyl, or a sugar substituent group and the other of X 1 and X 2 is hydroxyl, a protected hydroxyl, azido, or -O-J-SM;

J is a bifunctional linking moiety, selected from the group consisting of hydroxyl, acyl, amino, alkoxy, carboxy, benzyl, phenyl, nitro, thiol, thioalkoxy, halogen, alkyl, aryl, alkenyl and alkynyl; and

SM is a solid support medium.

2. The compound of claim 1 wherein Y 1 is S and Y 2 is BH 3 .

3. The compound of claim 1 wherein Y 3 is OH.

4. The compound of claim 1 wherein Bx is adenine, guanine, thymine, cytosine, or uracil.

5. A compound of formula I:

wherein:

Y 1 is O and Y 2 is BH 3 ; or

Y 1 is Se and Y 2 is OH;

Y 3 is NHR 1 , C 1 -C 12 alkyl, or C 1 -C 12 O-alkyl;

R 1 is hydrogen, substituted or unsubstituted C 1 -C 12 alkyl, substituted or unsubstituted C 2 -C 12 alkenyl, substituted or unsubstituted C 2 -C 12 alkynyl, substituted or unsubstituted C 5 -C 20 aralkyl, substituted or unsubstituted C 5 -C 20 alkylaryl, substituted or unsubstituted C 5 -C 20 heterocycloalkyl, substituted or unsubstituted C 5 -C 20 aryl, substituted or unsubstituted C 5 -C 20 heteroaryl, said substituted moieties comprising one or more substituents selected from hydrogen, halo, C 1 -C 12 alkyl, C 2 -C 12 alkenyl, C 2 -C 12 alkynyl, C 5 -C 20 aryl, C 1 -C 12 alkoxy, alkoxyalkyl, O-alkylaminoalkyl, O-alkylimidazolyl, S-alkyl, amino, alkylamino, dialkylamino, arylamino, diarylamino, C 5 -C 20 aryl, C 5 -C 20 heteroaryl, C 1 -C 12 cycloalkyl, C 1 -C 12 heterocyclolalkyl, O-aryl, S-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, nitro, cyano, aldehyde, carboxylic acid, hydroxy, alkylcarbonyl, aminocarbonyl, or mercapto;

Bx is an optionally protected heterocyclic base moiety;

one of X 1 and X 2 is H, hydroxyl, a protected hydroxyl, or a sugar substituent group and the other of X 1 and X 2 is hydroxyl, a protected hydroxyl, azido, or -O-J-SM;

J is a bifunctional linking moiety; selected from the group consisting of hydroxyl, acyl, amino, alkoxy, carboxy, benzyl, phenyl, nitro, thiol, thioalkoxy, halogen,alkyl, aryl, alkenyl and alkynyl; and

SM is a solid support medium.

6. A compound of formula I:

wherein:

Y 1 is Se and Y 2 is OH;

Y 3 is NHR 1 , OH, C 1 -C 12 alkyl, or C 1 -C 12 O-alkyl;

R 1 is hydrogen, substituted or unsubstituted C 1 -C 12 alkyl, substituted or unsubstituted C 2 -C 12 alkenyl, substituted or unsubstituted C 2 -C 12 alkynyl, substituted or unsubstituted C 5 -C 20 aralkyl substituted or unsubstituted C 5 -C 20 alkylaryl substituted or unsubstituted C 5 -C 20 heterocycloalkyl, substituted or unsubstituted C 5 -C 20 aryl, substituted or unsubstituted C 5 -C 20 heteroaryl, said substituted moieties comprising one or more substituents selected from hydrogen, halo, C 1 -C 12 alkyl, C 2 -C 12 alkenyl, C 2 -C 12 alkynyl, C 5 -C 20 aryl, C 1 -C 12 alkoxy, alkoxyalkyl, O-alkylaminoalkyl, O-alkylimidazolyl, S-alkyl, amino, alkylamino, dialkylamino, arylamino, diarylamino, C 5 -C 20 aryl, C 5 -C 20 heteroaryl, C 1 -C 12 cycloalkyl, C 1 -C 12 heterocyclolalkyl, O-aryl, S-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, nitro, cyano, aldehyde, carboxylic acid, hydroxy, alkylcarbonyl, aminocarbonyl, or mercapto;

Bx is an optionally protected heterocyclic base moiety;

one of X 1 and X 2 is H, hydroxyl, a protected hydroxyl, or a sugar substituent group and the other of X 1 and X 2 is hydroxyl, a protected hydroxyl, azido, or -O-J-SM;

J is a bifunctional linking moiety; and

SM is a solid support medium.

7. The compound of claim 6 wherein Y 3 is OH.

8. A compound of formula I:

wherein:

Y 1 is O and Y 2 is BH 3 ; or

Y 1 is Se and Y 2 is OH;

Y 3 is NHR 1 ;

R 1 is hydrogen substituted or unsubstituted C 1 -C 12 alkyl, substituted or unsubstituted C 2 -C 12 alkenyl, substituted or unsubstituted C 2 -C 12 alkynyl, substituted or unsubstituted C 5 -C 20 aralkyl, substituted or unsubstituted C 5 -C 20 alkylaryl, substituted or unsubstituted C 5 -C 20 heterocycloalkyl, substituted or unsubstituted C 5 -C 20 alkylaryl, substituted or unsubstituted C 5 -C 20 heteroaryl, said substituted moieties comprising one or more substituents selected from hydrogen, halo, C 1 -C 12 alkyl, C 2 -C 12 alkenyl, C 1 -C 12 alkynyl, C 5 -C 20 aryl, C 1 -C 12 alkoxy, alkoxyalkyl, O-alkylaminoalkyl, O-alkylimidazolyl, S-alkyl, amino, alkylamino, dialkylamino, arylamino, diarylamino, C 5 -C 20 aryl, C 5 -C 20 heteroaryl, C 1 -C 12 cycloalkyl, C 1 -C 1 heterocyclolalkyl, O-aryl, S-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, nitro, cyano, aldehyde, carboxylic acid, hydroxy, alkylcarbonyl, aminocarbonyl, or mercapto:

Bx is an optionally protected heterocyclic base moiety;

one of X 1 and X 2 is H, hydroxyl, a protected hydroxyl, or a sugar substituent group and the other of X 1 and X 2 is hydroxyl, a protected hydroxyl, azido, or -O-J-SM;

J is a bifunctional linking moiety; selected from the group consisting of hydroxyl, acyl, amino, alkoxy, carboxy, benzyl, phenyl, nitro, thiol, thioalkoxy, halogen, alkyl, aryl, alkenyl and alkynyl; and

SM is a solid support medium.

9. A compound of formula VIII:

wherein:

Y 1 is O or S;

Y 3 is NHR 1 OH, C 1 -C 12 alkyl, or C 1 -C 12 O-alkyl;

R 1 is hydrogen, substituted or unsubstituted C 1 -C 12 alkyl, substituted or unsubstituted C 2 -C 12 alkenyl, substituted or unsubstituted C 2 -C 12 alkynyl, substituted or unsubstituted C 5 -C 20 aralkyl, substituted or unsubstituted C 5 -C 20 alkylaryl, substituted or unsubstituted C 5 -C 20 heterocycloalkyl, substituted or unsubstituted C 5 -C 20 aryl, substituted or unsubstituted C 5 -C 20 heteroaryl, said substituted moieties comprising one or more substituents selected from hydrogen, halo, C 1 -C 12 alkyl, C 2 -C 12 alkenyl, C 2 -C 12 alkynyl, C 5 -C 20 aryl, C 1 -C 12 alkoxy, alkoxyalkyl, O-alkylaminoalkyl, O-alkylimidazolyl, S-alkyl, amino, alkylamino, dialkylamino, arylamino, diarylamino, C 5 -C 20 aryl, C 5 -C 20 heteroaryl, C 1 -C 12 cycloalkyl, C 1 -C 12 heterocyclolalkyl, O-aryl, S-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, nitro, cyano, aldehyde, carboxylic acid, hydroxy, alkylcarbonyl, aminocarbonyl, or mercapto; and

Bx is adenine, thymine, cytosine, guanine, or uracil.

10. The compound of claim 9 wherein Bx is thymine.

11. The compound of claim 9 wherein Y 1 is O.

12. A pharmaceutical formulation comprising a compound of claim 1 and a pharmaceutically acceptable excipient.

13. A pharmaceutically formulation comprising a compound of claim 5 and a pharmaceutically acceptable excipient.

14. A pharmaceutical formulation comprising a compound of claim 9 and a pharmaceutically acceptable excipient.

Assignments (2)
CHANGE OF NAME Recorded Jul 26, 2017
From: ISIS PHARMACEUTICALS, INC.
To: IONIS PHARMACEUTICALS, INC.
Reel/Frame 043341/0643 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 18, 2002
From: WYRZYKIEWICZ, TADEUSZ; COOK, PHILLIP DAN
To: ISIS PHARMACEUTICALS, INC.
Reel/Frame 013398/0391 →
Continuity (2)
Provisional Application 6030565200 · Jul 16, 2001
Related Publication 20030129615A1 · Jul 10, 2003