Patent US5998203A successfully refreshed from Google Patents.
US5998203A
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Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
Assignee: Sirna Therapeutics Inc
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Filing: 1996-04-16
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Granted: 1999-12-07
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Expires: 2016-04-16
Abstract
Abstract An enzymatic nucleic acid molecule comprising a 5'- and/or a 3'-cap structure, wherein said structure is not a 5'-5'-linked inverted nucleotide or a 3'-3'-linked inverted nucleotide.
Inventors
- 👤 Jasenka Matulic-Adamic
- 👤 Leonid Beigelman
- 👤 Alexander Karpeisky
- 👤 Thale Jarvis
- 👤 Nassim Usman
- 👤 Anthony DiRenzo
- 👤 Francine Wincott
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-11-05 11:12
CPC Classifications
C; C12; C12N; C12N15/00; C12N15/09; C12N15/11; C12N15/113; C07; C07H; C07H19/00; C07H19/02; C07H19/04; C07H19/06; C07H19/16; C07H21/00; C12N2310/00; C12N2310/10; C12N2310/12; C12N2310/121
Claims Analysis
Total Claims: 43
Claim 1
Independent Claim
129 words
An enzymatic nucleic acid molecule comprising a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure, wherein said 5'-cap structure is selected from the group consisting of 4',5'-methylene nucleotide; 1-(β-D-erythrofuranosyl)nucleotide; 4'-thio nucleotide, carbocyclic nucleotide; 5'-amino-alkyl phosphate; 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate; 6-aminohexyl phosphate; 12-aminododecyl phosphate; hydroxypropyl phosphate; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety; 1,4-butanediol phosphate; 5'-amino; and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; modified base nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide; 3'-3'-inverted abasic moiety; 3'-2'-inverted nucleotide moiety; 3'-2'-inverted abasic moiety; 1,4-butanediol; hexylphosphate; aminohexyl phosphate; 3'-phosphate; and a bridging methylphosphonate moiety.
Claim 2
Dependent Claim
129 words
An enzymatic nucleic acid molecule comprising a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure, wherein said 5'-cap structure is selected from the group consisting of 4',5'-methylene nucleotide; 1-(β-D-erythrofuranosyl)nucleotide; 4'-thio nucleotide, carbocyclic nucleotide; 5'-amino-alkyl phosphate; 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate; 6-aminohexyl phosphate; 12-aminododecyl phosphate; hydroxypropyl phosphate; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety; 1,4-butanediol phosphate; 5'-amino; and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; modified base nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide; 3'-3'-inverted abasic moiety; 3'-2'-inverted nucleotide moiety; 3'-2'-inverted abasic moiety; 1,4-butanediol; hexylphosphate; aminohexyl phosphate; 3'-phosphate; and a bridging methylphosphonate moiety.
Claim 3
Dependent Claim
19 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule is in a hammerhead motif.
Claim 4
Dependent Claim
31 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule is in a hairpin, hepatitis delta virus, group I intron, VS RNA or RNase P RNA motif.
Claim 5
Dependent Claim
18 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule comprises said 5'-cap structure.
Claim 6
Dependent Claim
18 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule comprises said 3'-cap structure.
Claim 7
Dependent Claim
22 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule comprises said 5'-cap structure and said 3'-cap structure.
Claim 8
Dependent Claim
16 words
The enzymatic nucleic acid molecule of claim 1, wherein said 5'- and 3'-cap structures are different.
Claim 9
Dependent Claim
20 words
The enzymatic nucleic acid molecule of claim 1, wherein said enzymatic nucleic acid molecule comprises at least one ribonucleotide residue.
Claim 10
Dependent Claim
59 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises: at least five ribose residues; at least three 5' terminal nucleotides are substituted with phosphorothioate linkages; position No. 4 of said enzymatic nucleic acid molecule is substituted with a 2'-C-allyl modification; and wherein said enzymatic nucleic acid molecule comprises at least ten 2'-O-methyl modifications.
Claim 11
Dependent Claim
54 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises: at least five ribose residues; a 2'-amino modification at position No. 4, at position No. 7, or at both position No. 4 and position No. 7; and wherein said enzymatic nucleic acid molecule comprises at least ten 2'-O-methyl modifications.
Claim 12
Dependent Claim
53 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises: at least five ribose residues; abasic substitution at position No. 4, at position No. 7, or at both position No. 4 and position No. 7; and wherein said enzymatic nucleic acid molecule comprises at least ten 2'-O-methyl modifications.
Claim 13
Dependent Claim
54 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises: at least five ribose residues; 6-methyl uridine substitutions at position No. 4, at position No. 7, or at both position No. 4 and position No. 7; and wherein said enzymatic nucleic acid molecule comprises at least ten 2'-O-methyl modifications.
Claim 14
Dependent Claim
53 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises at least five ribose residues; pyridin-4-one substitution at position No. 4, at position No. 7, or at both position No. 4 and position No. 7; and wherein said enzymatic nucleic acid molecule comprises at least ten 2'-O-methyl modifications.
Claim 15
Dependent Claim
28 words
The enzymatic nucleic acid molecule of claim 2, wherein said enzymatic nucleic acid molecule comprises a stem II region of length greater than or equal to two base-pairs.
Claim 16
Dependent Claim
29 words
The enzymatic nucleic acid molecule of claim 3, wherein said hairpin enzymatic nucleic acid molecule comprises a stem IV region of length greater than or equal to two base-pairs.
Claim 17
Dependent Claim
27 words
The enzymatic nucleic acid molecule of claim 3, wherein said hairpin enzymatic nucleic acid molecule comprises a stem II region of length between three and seven base-pairs.
Claim 18
Dependent Claim
17 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3'-3' linked inverted ribose.
Claim 19
Dependent Claim
17 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3'-2' linked inverted ribose.
Claim 20
Dependent Claim
17 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3'-2' linked inverted nucleotide.
Claim 21
Dependent Claim
17 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 5'-5' linked inverted ribose.
Claim 22
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 5'-amino group.
Claim 23
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 4'-thio nucleotide.
Claim 24
Dependent Claim
16 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 1,3-diamino-2-propyl phosphate group.
Claim 25
Dependent Claim
14 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a L-nucleotide.
Claim 26
Dependent Claim
14 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is an alpha-nucleotide.
Claim 27
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a threo-pentafuranosyl group.
Claim 28
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 3,4-dihydroxybutyl nucleotide.
Claim 29
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 3,5-dihydroxypentyl nucleotide.
Claim 30
Dependent Claim
14 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 1-(β-D-erythrofuranosyl)nucleotide.
Claim 31
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 4'-thio group.
Claim 32
Dependent Claim
14 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a L-nucleotide.
Claim 33
Dependent Claim
14 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is an alpha-nucleotide.
Claim 34
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3,4-dihydroxybutyl nucleotide.
Claim 35
Dependent Claim
15 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3,5-dihydroxypentyl nucleotide.
Claim 36
Dependent Claim
24 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 5'-amino group and said 3'-cap structure is a 3'-3'-inverted abasic nucleotide.
Claim 37
Dependent Claim
16 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3'-3'-inverted abasic nucleotide.
Claim 38
Dependent Claim
24 words
The enzymatic nucleic acid of claim 1, wherein said 5'-cap structure is a 5'-amino group and said 3'-cap structure is a 3'-2'-inverted abasic nucleotide.
Claim 39
Dependent Claim
16 words
The enzymatic nucleic acid of claim 1, wherein said 3'-cap structure is a 3'-2'-inverted abasic nucleotide.
Claim 40
Dependent Claim
12 words
A mammalian cell including the enzymatic nucleic acid molecule of claim 1.
Claim 41
Dependent Claim
14 words
The mammalian cell of claim 39, wherein said mammalian cell is a human cell.
Claim 42
Independent Claim
154 words
A method for producing an enzymatic nucleic acid molecule having activity to cleave an RNA or a single-stranded DNA molecule, comprising the step of forming said enzymatic nucleic acid molecule with a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure, wherein said 5'-cap structure is selected from the group consisting of 4',4'-methylene nucleotide; 1-(β-D-erythrofuranosyl)nucleotide; 4'-thio nucleotide, carbocyclic nucleotide; 5'-amino-alkyl phosphate; 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate; 6-aminohexyl phosphate; 12-aminododecyl phosphate; hydroxypropyl phosphate; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety; 1,4-butanediol phosphate; 5'-amino; and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; modified base nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide; 3'-3'-inverted abasic moiety; 3'-2'-inverted nucleotide moiety; 3'-2'-inverted abasic moiety; 1,4-butanediol; hexylphosphate; aminohexyl phosphate; 3'-phosphate; and a bridging methylphosphonate moiety.
Claim 43
Independent Claim
154 words
A method for producing an enzymatic nucleic acid molecule having activity to cleave an RNA or a single-stranded DNA molecule, comprising the step of forming said enzymatic nucleic acid molecule with a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure, wherein said 5'-cap structure is selected from the group consisting of 4',4'-methylene nucleotide; 1-(β-D-erythrofuranosyl)nucleotide; 4'-thio nucleotide, carbocyclic nucleotide; 5'-amino-alkyl phosphate; 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate; 6-aminohexyl phosphate; 12-aminododecyl phosphate; hydroxypropyl phosphate; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety; 1,4-butanediol phosphate; 5'-amino; and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide; 1,5-anhydrohexitol nucleotide; L-nucleotide; alpha-nucleotide; modified base nucleotide; threo-pentofuranosyl nucleotide; acyclic 3',4'-seco nucleotide; 3,4-dihydroxybutyl nucleotide; 3,5-dihydroxypentyl nucleotide; 3'-3'-inverted abasic moiety; 3'-2'-inverted nucleotide moiety; 3'-2'-inverted abasic moiety; 1,4-butanediol; hexylphosphate; aminohexyl phosphate; 3'-phosphate; and a bridging methylphosphonate moiety.
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| Annuity Stage | Window Opens | Payment Deadline | Surcharge Period Until | Estimated Fee | Status |
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| 3.5 Years (Year 4) | 2002-12-06 | 2003-06-07 | 2003-12-07 | $2000.00 | Window Closed / Lapsed |
| 7.5 Years (Year 8) | 2006-12-06 | 2007-06-07 | 2007-12-07 | $3760.00 | Window Closed / Lapsed |
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