Claims for Patent: 7,101,993

Summary for Patent: 7,101,993

Title:	Oligonucleotides containing 2′-O-modified purines
Abstract:	Compounds are provided containing purine nucleotides that bear moieties X at the 2′ position thereof wherein X is R1—(R2)n; R1 is C3-C20 alkyl, C4-C20 alkenyl or C2-C20 alkynyl; R2 is halogen, hydroxyl, thiol, keto, carboxyl, nitro, nitroso, nitrile, trifluoromethyl, trifluoromethoxy, O-alkyl, S-alkyl, NH-alkyl, N-dialkyl, O-aryl, S-aryl, NH-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, amino, N-phthalimido, imidazole, azido, hydrazino, hydroxylamino, isocyanato, sulfoxide, sulfone, sulfide, disulfide, silyl, aryl, heterocycle, carbocycle, intercalator, reporter molecule, conjugate, polyamine, polyamide, polyalkylene glycol, polyether, a group that enhances the pharmacodynamic properties of oligonucleotides, or a group that enhances the pharmacokinetic properties of oligonucleotides; and n is an integer from 0 to about 6. Such compounds are useful for modulating the synthesis of proteins.
Inventor(s):	Phillip Dan Cook, Daniel Peter Claude McGee, Charles John Guinosso
Assignee:	Ionis Pharmaceuticals Inc
Application Number:	US07/967,267
Patent Claims:	1. A compound having the structure: wherein X is R1—(R2)n; R1 is C2-C20 alkyl, C2-C20 alkenyl, or C2-C20 alkynyl and n is 1 to 6; R2 is halogen, nitro, nitroso, nitrile, trifluoromethyl, trifluoromethoxy, O-alkyl, S-alkyl, NH-alkyl, N-dialkyl, O-aryl, S-aryl, NH-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, imidazole, N-phthalimido, azido, hydrazino, hydroxylamino, isocyanato, sulfoxide, sulfone, sulfide, disulfide, silyl, aryl, heterocycle, carbocycle, intercalator, reporter molecule, conjugate, polyamine, polyamide, polyalkylene glycol, or polyether; and either one of T3 and T5 is OH, a hydroxyl blocking group, phosphate or an activated phosphate group and the other of T3 and T5 is a nucleotide or both T3 and T5 are nucleotides. 2. A compound having the structure: wherein X is R1—(R2)n; R1 is C1-C20 alkyl, C4-C20 alkenyl, or C2-C20 alkynyl; R2 is halogen, nitro, nitroso, nitrile, trifluoromethyl, trifluoromethoxy, O-alkyl, S-alkyl, NH-alkyl, N-dialkyl, O-aryl, S-aryl, NH-aryl, O-aralkyl, S-aralkyl, NH-aralkyl, imidazole, N-phthalimido, azido, hydrazino, hydroxylamino, isocyanato, sulfoxide, sulfone, sulfide, disulfide, silyl, aryl, heterocycle, carbocycle, intercalator, reporter molecule, conjugate, polyamine, polyamide, polyalkylene glycol, or polyether; either one of T3 and T5 is OH, a hydroxyl blocking group, phosphate or an activated phosphate group and the other of T3 and T5 is a nucleotide, or both T3 and T5 are nucleotides; and n is an integer from 1 to 6. 3. The compound of claim 1 wherein R1 is C4-C20 alkyl. 4. The compound of claim 1 wherein R1 is C5-C20 alkyl. 5. The compound of claim 1 wherein R1 is C3-C20 alkyl and n is 1. 6. The compound of claim 1 wherein R1 is C4-C20 alkenyl and n is 1. 7. The compound of claim 1 wherein R1 is C2-C20 alkynyl and n is 1. 8. The compound of claim 1 wherein R1 is C2-C20 alkyl and n is 1 to 6. 9. The compound of claim 1 wherein R1 is C2-C20 alkenyl and n is 1 to 6. 10. The compound of claim wherein R1 is C2-C20 alkynyl and n is 1 to 6. 11. The compound of claim 2 wherein R1 is C1-C20 alkyl. 12. The compound of claim 2 wherein R1 is C4-C20 alkenyl. 13. The compound of claim 2 wherein R1 is C2-C20 alkynyl. 14. A compound having the structure: wherein X is R1—(R2)n; R1 is C4-C20 alkyl, C4-C20 alkenyl, or C2-C20 alkynyl and n is 0; or R1 is C1-C20 alkyl, C2-C20 alkenyl, or C2-C20 alkynyl and n is 1 to 6; R2 is hydroxyl, thiol, keto, carboxyl, or amino; and either one of T3 and T5 is OH, a hydroxyl blocking group, phosphate or an activated phosphate group and the other of T3 and T5 is a nucleotide or both T3 and T5 are nucleotides. 15. A compound having the structure: wherein X is R1—(R2)n; R1 is C1-C20 alkyl, C4-C20 alkenyl, or C2-C20 alkynyl; R2 is hydroxyl, thiol, keto, carboxyl, or amino; either one of T3 and T5 is OH, a hydroxyl blocking group, phosphate or an activated phosphate group and the other of T3 and T5 is a nucleotide, or both T3 and T5 are nucleotides; and n is an integer from 0 to 6.