AU2009307677C1 - Compositions and methods for inhibiting expression of transthyretin - Google Patents

Compositions and methods for inhibiting expression of transthyretin Download PDF

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Publication number
AU2009307677C1
AU2009307677C1 AU2009307677A AU2009307677A AU2009307677C1 AU 2009307677 C1 AU2009307677 C1 AU 2009307677C1 AU 2009307677 A AU2009307677 A AU 2009307677A AU 2009307677 A AU2009307677 A AU 2009307677A AU 2009307677 C1 AU2009307677 C1 AU 2009307677C1
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Australia
Prior art keywords
dsrna
ttr
nucleotide
seq
human
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AU2009307677A
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AU2009307677A1 (en
AU2009307677B2 (en
Inventor
Rene Alvarez
Qingmin Chen
Gregory Hinkle
Stuart Milstein
Dinah Wen-Yee Sah
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2009307677B2 publication Critical patent/AU2009307677B2/en
Priority to AU2015249072A priority Critical patent/AU2015249072C1/en
Priority to AU2017225110A priority patent/AU2017225110B2/en
Priority to AU2019216630A priority patent/AU2019216630A1/en
Priority to AU2021203272A priority patent/AU2021203272B2/en
Publication of AU2009307677C1 publication Critical patent/AU2009307677C1/en
Priority to AU2023248138A priority patent/AU2023248138B2/en
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    • 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
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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
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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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    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • 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
    • C12N2310/10—Type of nucleic acid
    • 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
    • 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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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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AU2009307677A 2008-10-20 2009-10-20 Compositions and methods for inhibiting expression of transthyretin Active 2034-10-20 AU2009307677C1 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU2015249072A AU2015249072C1 (en) 2008-10-20 2015-10-28 Compositions and methods for inhibiting expression of transthyretin
AU2017225110A AU2017225110B2 (en) 2008-10-20 2017-09-08 Compositions and methods for inhibiting expression of transthyretin
AU2019216630A AU2019216630A1 (en) 2008-10-20 2019-08-14 Compositions and methods for inhibiting expression of transthyretin
AU2021203272A AU2021203272B2 (en) 2008-10-20 2021-05-21 Compositions and methods for inhibiting expression of transthyretin
AU2023248138A AU2023248138B2 (en) 2008-10-20 2023-10-12 Compositions and methods for inhibiting expression of transthyretin

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US10695608P 2008-10-20 2008-10-20
US61/106,956 2008-10-20
US11573808P 2008-11-18 2008-11-18
US61/115,738 2008-11-18
US15667009P 2009-03-02 2009-03-02
US61/156,670 2009-03-02
US18554509P 2009-06-09 2009-06-09
US61/185,545 2009-06-09
US24278309P 2009-09-15 2009-09-15
US61/242,783 2009-09-15
US24479409P 2009-09-22 2009-09-22
US61/244,794 2009-09-22
PCT/US2009/061381 WO2010048228A2 (en) 2008-10-20 2009-10-20 Compositions and methods for inhibiting expression of transthyretin

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2015249072A Division AU2015249072C1 (en) 2008-10-20 2015-10-28 Compositions and methods for inhibiting expression of transthyretin

Publications (3)

Publication Number Publication Date
AU2009307677A1 AU2009307677A1 (en) 2010-04-29
AU2009307677B2 AU2009307677B2 (en) 2015-07-30
AU2009307677C1 true AU2009307677C1 (en) 2022-09-22

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AU2009307677A Active 2034-10-20 AU2009307677C1 (en) 2008-10-20 2009-10-20 Compositions and methods for inhibiting expression of transthyretin

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US (9) US8168775B2 (enExample)
EP (4) EP2937418B1 (enExample)
JP (7) JP5791505B2 (enExample)
KR (6) KR102578331B1 (enExample)
CN (3) CN106834291B (enExample)
AU (1) AU2009307677C1 (enExample)
CA (3) CA3222620A1 (enExample)
CY (3) CY1116944T1 (enExample)
DK (2) DK2344639T3 (enExample)
EA (3) EA036772B1 (enExample)
ES (2) ES2543004T3 (enExample)
HR (2) HRP20150796T1 (enExample)
HU (3) HUE037875T2 (enExample)
IL (5) IL310600A (enExample)
LT (2) LT2937418T (enExample)
LU (1) LUC00098I2 (enExample)
MX (4) MX360460B (enExample)
NL (1) NL300965I2 (enExample)
NO (2) NO2937418T3 (enExample)
NZ (2) NZ602404A (enExample)
PL (2) PL2937418T3 (enExample)
PT (2) PT2937418T (enExample)
SG (3) SG10201703242XA (enExample)
SI (2) SI2937418T1 (enExample)
SM (2) SMT201800027T1 (enExample)
WO (1) WO2010048228A2 (enExample)
ZA (1) ZA201102876B (enExample)

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