HRP20150796T1 - Pripravci i postupci za inhibiciju ekspresije transtiretina - Google Patents

Pripravci i postupci za inhibiciju ekspresije transtiretina Download PDF

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Publication number
HRP20150796T1
HRP20150796T1 HRP20150796TT HRP20150796T HRP20150796T1 HR P20150796 T1 HRP20150796 T1 HR P20150796T1 HR P20150796T T HRP20150796T T HR P20150796TT HR P20150796 T HRP20150796 T HR P20150796T HR P20150796 T1 HRP20150796 T1 HR P20150796T1
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HR
Croatia
Prior art keywords
dsrna
nucleotide
ttr
antisense strand
transthyretin
Prior art date
Application number
HRP20150796TT
Other languages
English (en)
Croatian (hr)
Inventor
Dinah Wen-Yee 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 HRP20150796T1 publication Critical patent/HRP20150796T1/hr

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    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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
    • A—HUMAN NECESSITIES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • 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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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—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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    • C12N15/00—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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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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
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    • C12N2310/32—Chemical structure of the sugar
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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HRP20150796TT 2008-10-20 2009-10-20 Pripravci i postupci za inhibiciju ekspresije transtiretina HRP20150796T1 (hr)

Applications Claiming Priority (8)

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
EP09810834.3A EP2344639B1 (en) 2008-10-20 2009-10-20 Compositions and methods for inhibiting expression of transthyretin

Publications (1)

Publication Number Publication Date
HRP20150796T1 true HRP20150796T1 (hr) 2015-09-11

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Application Number Title Priority Date Filing Date
HRP20150796TT HRP20150796T1 (hr) 2008-10-20 2009-10-20 Pripravci i postupci za inhibiciju ekspresije transtiretina
HRP20180093TT HRP20180093T1 (hr) 2008-10-20 2018-01-17 Pripravci i postupci za inhibiciju ekspresije transtiretina

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Application Number Title Priority Date Filing Date
HRP20180093TT HRP20180093T1 (hr) 2008-10-20 2018-01-17 Pripravci i postupci za inhibiciju ekspresije transtiretina

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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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