AU2020364248A1 - Modified oligonucleotides - Google Patents

Modified oligonucleotides Download PDF

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Publication number
AU2020364248A1
AU2020364248A1 AU2020364248A AU2020364248A AU2020364248A1 AU 2020364248 A1 AU2020364248 A1 AU 2020364248A1 AU 2020364248 A AU2020364248 A AU 2020364248A AU 2020364248 A AU2020364248 A AU 2020364248A AU 2020364248 A1 AU2020364248 A1 AU 2020364248A1
Authority
AU
Australia
Prior art keywords
nucleotide
antisense strand
double
stranded rna
dsrna
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2020364248A
Other languages
English (en)
Inventor
Dhrubajyoti Datta
Pawan Kumar
Muthiah Manoharan
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
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 AU2020364248A1 publication Critical patent/AU2020364248A1/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2020364248A 2019-10-11 2020-10-13 Modified oligonucleotides Pending AU2020364248A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962914010P 2019-10-11 2019-10-11
US62/914,010 2019-10-11
PCT/US2020/055301 WO2021072395A1 (en) 2019-10-11 2020-10-13 Modified oligonucleotides

Publications (1)

Publication Number Publication Date
AU2020364248A1 true AU2020364248A1 (en) 2022-04-14

Family

ID=75436777

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020364248A Pending AU2020364248A1 (en) 2019-10-11 2020-10-13 Modified oligonucleotides

Country Status (6)

Country Link
US (2) US12497613B2 (https=)
EP (1) EP4041248A4 (https=)
JP (2) JP7761558B2 (https=)
AU (1) AU2020364248A1 (https=)
CA (1) CA3157486A1 (https=)
WO (1) WO2021072395A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11504391B1 (en) 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
WO2019217397A2 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
US20230407302A1 (en) * 2022-02-04 2023-12-21 New York University Novel efficacious microrna-30c analogs reduce apolipoprotein b secretion in human liver cells

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10133858A1 (de) * 2001-07-12 2003-02-06 Aventis Pharma Gmbh Synthetische doppelsträngige Oligonucleotide zur gezielten Hemmung der Genexpression
WO2006047842A2 (en) * 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
WO2008025160A1 (en) * 2006-08-31 2008-03-06 Mcgill University Oxepane nucleosides and oligonucleotides, uses thereof and methods of making the same
JP2009215241A (ja) * 2008-03-11 2009-09-24 Gifu Univ ピラノース環を有するヌクレオシド類似体、核酸類似体及び核酸
WO2013033230A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use

Also Published As

Publication number Publication date
EP4041248A4 (en) 2024-03-20
US20260055401A1 (en) 2026-02-26
US20220389419A1 (en) 2022-12-08
US12497613B2 (en) 2025-12-16
JP2022550979A (ja) 2022-12-06
JP7761558B2 (ja) 2025-10-28
WO2021072395A1 (en) 2021-04-15
CA3157486A1 (en) 2021-04-15
JP2026002913A (ja) 2026-01-08
EP4041248A1 (en) 2022-08-17

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