Non-nucleotide containing RNA

• US 6,849,726 B2
• Filed: 03/17/2003
• Issued: 02/01/2005
• Est. Priority Date: 09/02/1993
• Status: Expired due to Fees

• Current Assignee: Sirna Therapeutics, Inc. (Alnylam Pharmaceuticals Incorporated)
• Original Assignee: Sirna Therapeutics, Inc. (Alnylam Pharmaceuticals Incorporated)
• Inventors: Wincott, Francine, Karpeisky, Alex, Beigelman, Leonid, Matulic-Adamic, Jasenka, Usman, Nassim
• Primary Examiner(s): Riley, Jezia
• Application Number:
  US10/390,206
• Publication Number:
  US 20030165969A1
• Time in Patent Office: 687 Days
• Field of Search: 536/22.1, 536/23.1, 536/25.3, 536/25.31, 435/6
• US Class Current: 536/23.1
• CPC Class Codes: C07H 19/06 Pyrimidine radicals
• CPC Class Codes: C07H 19/10 with the saccharide radical esterified by phosphoric or polyphosphoric acids
• CPC Class Codes: C07H 19/16 Purine radicals
• CPC Class Codes: C07H 19/20 with the saccharide radical esterified by phosphoric or polyphosphoric acids
• CPC Class Codes: C07H 21/00 Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
• CPC Class Codes: C12N 15/111 General methods applicable to biologically active non-coding nucleic acids
• CPC Class Codes: C12N 15/113 Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing when used in plants C12N15/8218
• CPC Class Codes: C12N 15/1137 against enzymes viral enzymes C12N15/1131; receptors C12N15/1138
• CPC Class Codes: C12N 15/1138 against receptors or cell surface proteins
• CPC Class Codes: C12N 2310/121 Hammerhead
• CPC Class Codes: C12N 2310/315 Phosphorothioates
• CPC Class Codes: C12N 2310/318 where the PO2 is completely replaced, e.g. MMI or formacetal
• CPC Class Codes: C12N 2310/32 of the sugar
• CPC Class Codes: C12N 2310/321 2'-O-R Modification
• CPC Class Codes: C12N 2310/322 2'-R Modification
• CPC Class Codes: C12N 2310/33 of the base
• CPC Class Codes: C12N 2310/332 Abasic residue
• CPC Class Codes: C12N 2310/346 having a combination of backbone and sugar modifications
• CPC Class Codes: C12N 2310/3521 Methyl
• CPC Class Codes: C12N 2330/30 chemically synthesised
• CPC Class Codes: C12Y 304/24017 : Stromelysin 1 (3.4.24.17)

Abstract

The present invention relates to RNA molecules containing one or more non-nucleotide mimetics.

Claims

1. A double stranded RNA molecule comprising at least one of the non-nucleotide moieties selected from the group consisting of:

wherein, said “

n”

is an integer of between 1 and 10;

X is independently oxygen, nitrogen, sulfur or substituted carbons including alkyl or alkene;

R is independently an H, alkyl, alkenyl or alkynyl of 1-10 carbon atoms; and

Y is independently a phosphodiester, ether or amide linkage to the nucleic acid molecule.

3. The double stranded RNA molecule of claim 1, wherein said RNA comprises one or more chemical modifications selected from nucleic acid sugar, base, and backbone modifications, including any combination thereof.

5. The double stranded RNA molecule of claim 1, wherein said non-nucleotide covalently links one strand of said molecule to another strand of said molecule.

7. The double stranded RNA molecule of claim 3, wherein said nucleic acid sugar modification is selected from 2′

-deoxy, 2′

-O-methyl, 2′

-amino, and 2′

-fluoro.

8. The double stranded RNA molecule of claim 3, wherein said nucleic acid backbone modification comprises phosphorothioate.

9. The double stranded RNA molecule of claim 3, wherein said nucleic acid base modification is selected from aminopurine, hypoxanthine, and imidazoyl.

2. A double stranded RNA molecule comprising at least one non-nucleotide moiety, wherein said non-nucleotide moiety lacks a nucleic acid base and wherein said non-nucleotide comprises a nucleotide sugar and phosphate component.

4. The double stranded RNA molecule of claim 2, wherein said RNA comprises one or more chemical modifications selected from nucleic acid sugar, base, and backbone modifications, including any combination thereof.

6. The double stranded RNA molecule of claim 2, wherein said non-nucleotide covalently links one strand of said molecule to another strand of said molecule.

10. The double stranded RNA molecule of claim 4, wherein said nucleic acid sugar modification is selected from 2′

-deoxy, 2′

-O-methyl, 2′

-amino, and 2′

-fluoro.

11. The double stranded RNA molecule of claim 4, wherein said nucleic acid backbone modification comprises phosphorothioate.

12. The double stranded RNA molecule of claim 4, wherein said nucleic acid base modification is selected from aminopurine, hypoxanthine, and imidazoyl.