ZA201102876B - Compositions and methods for inhibiting expression of transthyretin - Google Patents

Compositions and methods for inhibiting expression of transthyretin

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Publication number
ZA201102876B
ZA201102876B ZA2011/02876A ZA201102876A ZA201102876B ZA 201102876 B ZA201102876 B ZA 201102876B ZA 2011/02876 A ZA2011/02876 A ZA 2011/02876A ZA 201102876 A ZA201102876 A ZA 201102876A ZA 201102876 B ZA201102876 B ZA 201102876B
Authority
ZA
South Africa
Prior art keywords
transthyretin
compositions
methods
inhibiting expression
inhibiting
Prior art date
Application number
ZA2011/02876A
Other languages
English (en)
Inventor
Dinah Wenyee Sah
Gregory Hinkle
Rene Alvarez
Stuart Milstein
Qingmin Chen
Original Assignee
Alnylam Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of ZA201102876B publication Critical patent/ZA201102876B/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6527—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07F9/6533—Six-membered rings
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    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/32—Special delivery means, e.g. tissue-specific

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ZA2011/02876A 2008-10-20 2011-04-18 Compositions and methods for inhibiting expression of transthyretin ZA201102876B (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US10695608P 2008-10-20 2008-10-20
US11573808P 2008-11-18 2008-11-18
US15667009P 2009-03-02 2009-03-02
US18554509P 2009-06-09 2009-06-09
US24278309P 2009-09-15 2009-09-15
US24479409P 2009-09-22 2009-09-22
PCT/US2009/061381 WO2010048228A2 (en) 2008-10-20 2009-10-20 Compositions and methods for inhibiting expression of transthyretin

Publications (1)

Publication Number Publication Date
ZA201102876B true ZA201102876B (en) 2015-06-24

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ZA2011/02876A ZA201102876B (en) 2008-10-20 2011-04-18 Compositions and methods for inhibiting expression of transthyretin

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US (9) US8168775B2 (cg-RX-API-DMAC7.html)
EP (4) EP2937418B1 (cg-RX-API-DMAC7.html)
JP (7) JP5791505B2 (cg-RX-API-DMAC7.html)
KR (6) KR101635436B1 (cg-RX-API-DMAC7.html)
CN (3) CN106834291B (cg-RX-API-DMAC7.html)
AU (1) AU2009307677C1 (cg-RX-API-DMAC7.html)
CA (3) CA3222620A1 (cg-RX-API-DMAC7.html)
CY (3) CY1116944T1 (cg-RX-API-DMAC7.html)
DK (2) DK2937418T3 (cg-RX-API-DMAC7.html)
EA (3) EA036772B1 (cg-RX-API-DMAC7.html)
ES (2) ES2656516T3 (cg-RX-API-DMAC7.html)
HR (2) HRP20150796T1 (cg-RX-API-DMAC7.html)
HU (3) HUE037875T2 (cg-RX-API-DMAC7.html)
IL (5) IL281434B2 (cg-RX-API-DMAC7.html)
LT (2) LT2937418T (cg-RX-API-DMAC7.html)
LU (1) LUC00098I2 (cg-RX-API-DMAC7.html)
MX (4) MX360460B (cg-RX-API-DMAC7.html)
NL (1) NL300965I2 (cg-RX-API-DMAC7.html)
NO (2) NO2937418T3 (cg-RX-API-DMAC7.html)
NZ (2) NZ592867A (cg-RX-API-DMAC7.html)
PL (2) PL2937418T3 (cg-RX-API-DMAC7.html)
PT (2) PT2344639E (cg-RX-API-DMAC7.html)
SG (3) SG10201912276XA (cg-RX-API-DMAC7.html)
SI (2) SI2344639T1 (cg-RX-API-DMAC7.html)
SM (2) SMT201800027T1 (cg-RX-API-DMAC7.html)
WO (1) WO2010048228A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201102876B (cg-RX-API-DMAC7.html)

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