CN117965536A - 寡核苷酸组合物及其方法 - Google Patents

寡核苷酸组合物及其方法 Download PDF

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Publication number
CN117965536A
CN117965536A CN202311216358.3A CN202311216358A CN117965536A CN 117965536 A CN117965536 A CN 117965536A CN 202311216358 A CN202311216358 A CN 202311216358A CN 117965536 A CN117965536 A CN 117965536A
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oligonucleotides
composition
oligonucleotide
linkages
modified
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Chinese (zh)
Inventor
马里亚·D·弗兰克-卡梅涅茨基
杨海林
阿伦·莫里斯
钱德拉·瓦尔格赛
克里斯托弗·J·弗朗西斯
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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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
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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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    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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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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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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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
    • C12N2310/3525—MOE, methoxyethoxy

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  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN202311216358.3A 2015-07-22 2016-07-22 寡核苷酸组合物及其方法 Pending CN117965536A (zh)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201562195779P 2015-07-22 2015-07-22
US62/195,779 2015-07-22
US201562236847P 2015-10-02 2015-10-02
US62/236,847 2015-10-02
US201662331960P 2016-05-04 2016-05-04
US62/331,960 2016-05-04
CN201680042413.3A CN108135921B (zh) 2015-07-22 2016-07-22 寡核苷酸组合物及其方法
PCT/US2016/043598 WO2017015575A1 (en) 2015-07-22 2016-07-22 Oligonucleotide compositions and methods thereof

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CN201680042413.3A Active CN108135921B (zh) 2015-07-22 2016-07-22 寡核苷酸组合物及其方法
CN201680043299.6A Pending CN108025089A (zh) 2015-07-22 2016-07-22 寡核苷酸组合物及其方法

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US (6) US20180216107A1 (enExample)
EP (3) EP4344744A3 (enExample)
JP (7) JP2018525357A (enExample)
KR (1) KR20180028516A (enExample)
CN (3) CN117965536A (enExample)
AU (3) AU2016297155B2 (enExample)
BR (1) BR112017028636A2 (enExample)
CA (1) CA2989682A1 (enExample)
CL (1) CL2018000145A1 (enExample)
CO (1) CO2018001484A2 (enExample)
CR (1) CR20180107A (enExample)
EC (1) ECSP18010463A (enExample)
HK (2) HK1255369A1 (enExample)
IL (2) IL320434A (enExample)
MA (1) MA43072A (enExample)
MX (2) MX2018000796A (enExample)
NI (1) NI201800009A (enExample)
PE (1) PE20181174A1 (enExample)
PH (1) PH12018500145A1 (enExample)
SG (1) SG10201912900XA (enExample)
SV (1) SV2018005619A (enExample)
TW (1) TW201707711A (enExample)
WO (2) WO2017015575A1 (enExample)

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