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MX2020010802A - Moleculas inhibidoras de acidos nucleicos de caracter bicatenario modificadas con nucleotidos que aumentan la tm. - Google Patents

Moleculas inhibidoras de acidos nucleicos de caracter bicatenario modificadas con nucleotidos que aumentan la tm.

Info

Publication number
MX2020010802A
MX2020010802A MX2020010802A MX2020010802A MX2020010802A MX 2020010802 A MX2020010802 A MX 2020010802A MX 2020010802 A MX2020010802 A MX 2020010802A MX 2020010802 A MX2020010802 A MX 2020010802A MX 2020010802 A MX2020010802 A MX 2020010802A
Authority
MX
Mexico
Prior art keywords
sub
double
nucleic acid
stranded nucleic
acid inhibitor
Prior art date
Application number
MX2020010802A
Other languages
English (en)
Inventor
Naim Nazef
Weimin Wang
Bob Dale Brown
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2020010802A publication Critical patent/MX2020010802A/es

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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
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1137—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 against enzymes
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    • C12Y—ENZYMES
    • C12Y102/00—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003—Aldehyde dehydrogenase (NAD+) (1.2.1.3)
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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
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • 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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    • C12N2310/30—Chemical structure
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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
    • C12N2310/531—Stem-loop; Hairpin
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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
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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
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9

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

Abstract

En la presente se proporcionan moléculas inhibidoras de ácidos nucleicos de carácter bicatenario que tienen una cadena codificante con una estructura de tallo-bucle y una cadena no codificante, donde la parte del tallo de la estructura de tallo-bucle contiene uno o más nucleótidos que aumentan la Tm, también se proporcionan métodos y composiciones para reducir la expresión de genes diana y métodos y composiciones para tratar una enfermedad de interés.
MX2020010802A 2018-04-13 2019-04-11 Moleculas inhibidoras de acidos nucleicos de caracter bicatenario modificadas con nucleotidos que aumentan la tm. MX2020010802A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862657428P 2018-04-13 2018-04-13
US201862778755P 2018-12-12 2018-12-12
PCT/US2019/027021 WO2019200124A1 (en) 2018-04-13 2019-04-11 Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides

Publications (1)

Publication Number Publication Date
MX2020010802A true MX2020010802A (es) 2020-10-28

Family

ID=68161339

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020010802A MX2020010802A (es) 2018-04-13 2019-04-11 Moleculas inhibidoras de acidos nucleicos de caracter bicatenario modificadas con nucleotidos que aumentan la tm.

Country Status (10)

Country Link
US (1) US11873488B2 (es)
EP (1) EP3773609A4 (es)
JP (1) JP2021520355A (es)
KR (1) KR20200144100A (es)
CN (1) CN111971051A (es)
AU (1) AU2019252529A1 (es)
CA (1) CA3094008A1 (es)
IL (1) IL277813A (es)
MX (1) MX2020010802A (es)
WO (1) WO2019200124A1 (es)

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MX2021007001A (es) * 2018-12-12 2021-10-13 Dicerna Pharmaceuticals Inc Moleculas inhibidoras de acido nucleico de cadena doble que contienen un tribucle.
EP3963072A1 (en) * 2019-05-03 2022-03-09 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
JP2024518564A (ja) * 2021-05-11 2024-05-01 ディセルナ ファーマシューティカルズ インコーポレイテッド 中枢神経系のニューロンを標的とするための脂質コンジュゲーション
WO2023069707A2 (en) * 2021-10-22 2023-04-27 Sirnaomics, Inc. Products and compositions
TW202400786A (zh) * 2022-03-16 2024-01-01 日商第一三共股份有限公司 具有RNAi活性的化學修飾寡核苷酸

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US8273866B2 (en) * 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
WO2009117589A1 (en) 2008-03-21 2009-09-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucleosides and methods for their use
AU2009293636A1 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsRNA possessing modifications
US20110288147A1 (en) 2008-09-22 2011-11-24 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop
EP2379083B1 (en) * 2008-12-18 2017-08-16 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
KR101791702B1 (ko) * 2009-04-03 2017-10-30 다이서나 파마수이티컬, 인크. 비대칭 이중가닥 ｒｎａ에 의한 ｋｒａｓ의 특이적 저해를 위한 방법 및 조성물
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KR20150131365A (ko) * 2013-03-15 2015-11-24 미라젠 세러퓨틱스 인코포레이티드 브리지드 바이사이클릭 뉴클레오시드
JP2016528887A (ja) * 2013-07-03 2016-09-23 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物
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EP3645013A4 (en) * 2017-06-29 2021-07-21 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF HMGB1

Also Published As

Publication number Publication date
CN111971051A (zh) 2020-11-20
EP3773609A1 (en) 2021-02-17
US20190316125A1 (en) 2019-10-17
US11873488B2 (en) 2024-01-16
KR20200144100A (ko) 2020-12-28
JP2021520355A (ja) 2021-08-19
IL277813A (en) 2020-11-30
WO2019200124A1 (en) 2019-10-17
EP3773609A4 (en) 2022-05-18
CA3094008A1 (en) 2019-10-17
AU2019252529A1 (en) 2020-10-01

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