CA2958758A1 - Modified double-stranded rna agents - Google Patents

Modified double-stranded rna agents Download PDF

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Publication number
CA2958758A1
CA2958758A1 CA2958758A CA2958758A CA2958758A1 CA 2958758 A1 CA2958758 A1 CA 2958758A1 CA 2958758 A CA2958758 A CA 2958758A CA 2958758 A CA2958758 A CA 2958758A CA 2958758 A1 CA2958758 A1 CA 2958758A1
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Canada
Prior art keywords
dsrna agent
antisense strand
nucleotide
strand
nucleotides
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Pending
Application number
CA2958758A
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English (en)
French (fr)
Inventor
Martin Maier
Don Foster
Stuart Milstein
Satya Kuchimanchi
Vasant Jadhav
Kallanthottathil Rajeev
Muthiah Manoharan
Rubina PARMAR
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CA2958758A1 publication Critical patent/CA2958758A1/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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Animal Behavior & Ethology (AREA)
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  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
CA2958758A 2014-08-20 2015-08-14 Modified double-stranded rna agents Pending CA2958758A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462039507P 2014-08-20 2014-08-20
US62/039,507 2014-08-20
US201462083744P 2014-11-24 2014-11-24
US62/083,744 2014-11-24
US201462093919P 2014-12-18 2014-12-18
US62/093,919 2014-12-18
PCT/US2015/045407 WO2016028649A1 (en) 2014-08-20 2015-08-14 Modified double-stranded rna agents

Publications (1)

Publication Number Publication Date
CA2958758A1 true CA2958758A1 (en) 2016-02-25

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ID=53969461

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2958758A Pending CA2958758A1 (en) 2014-08-20 2015-08-14 Modified double-stranded rna agents

Country Status (12)

Country Link
US (11) US10233448B2 (enExample)
EP (4) EP3808846A1 (enExample)
JP (5) JP7289610B2 (enExample)
KR (3) KR20240010762A (enExample)
CN (3) CN107075516A (enExample)
CA (1) CA2958758A1 (enExample)
EA (1) EA201790420A1 (enExample)
IL (3) IL316808A (enExample)
MX (3) MX2017002144A (enExample)
NZ (1) NZ730296A (enExample)
SG (3) SG11201701166UA (enExample)
WO (1) WO2016028649A1 (enExample)

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