BR112018008344A2 - molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição - Google Patents

molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição

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Publication number
BR112018008344A2
BR112018008344A2 BR112018008344A BR112018008344A BR112018008344A2 BR 112018008344 A2 BR112018008344 A2 BR 112018008344A2 BR 112018008344 A BR112018008344 A BR 112018008344A BR 112018008344 A BR112018008344 A BR 112018008344A BR 112018008344 A2 BR112018008344 A2 BR 112018008344A2
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Brazil
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composition
disease
ameliorating
vector
treating
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BR112018008344A
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English (en)
Inventor
Cina Cima
Zheng Jane
Harborth Jens
Minomi Kenjirou
Vaish Narendra
Ying Wenbin
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Nitto Denko Corp
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Publication of BR112018008344A2 publication Critical patent/BR112018008344A2/pt

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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
    • 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
    • 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
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/01018—Glutathione transferase (2.5.1.18)
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

esta invenção provê compostos, compos¬ições e métodos para modular a expressão de genes alvo usando interferência de rna. as estruturas de rnai e moléculas desta invenção podem ser usadas para modular ou silenciar a expressão de genes, com níveis altos de atividade de rnai e ações alvo reduzidas. estruturas vantajosas incluem sirnas alvejadas a qualquer gene tendo um ou mais 2?-desóxi nucleotídeos localizados na região de semente. as moléculas de interferência de rna podem ser usadas em métodos para prevenir ou tratar doenças.
BR112018008344A 2015-12-13 2016-12-12 molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição BR112018008344A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562266675P 2015-12-13 2015-12-13
PCT/US2016/066239 WO2017106111A1 (en) 2015-12-13 2016-12-12 Sirna structures for high activity and reduced off target

Publications (1)

Publication Number Publication Date
BR112018008344A2 true BR112018008344A2 (pt) 2018-10-30

Family

ID=59018392

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018008344A BR112018008344A2 (pt) 2015-12-13 2016-12-12 molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição

Country Status (12)

Country Link
US (3) US10358647B2 (pt)
EP (2) EP3816287A1 (pt)
JP (1) JP6457704B2 (pt)
KR (1) KR20180086260A (pt)
CN (1) CN108367022A (pt)
AU (1) AU2016371624B2 (pt)
BR (1) BR112018008344A2 (pt)
CA (1) CA3005937C (pt)
ES (1) ES2865030T3 (pt)
PT (1) PT3386519T (pt)
TW (1) TWI752927B (pt)
WO (1) WO2017106111A1 (pt)

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CN110714025A (zh) * 2018-07-13 2020-01-21 中国农业大学 一种靶向猪ttn基因的打靶载体及其应用
AU2019347774A1 (en) 2018-09-28 2021-03-25 Nutcracker Therapeutics, Inc. Lipid nanoparticle formulations comprising lipidated cationic peptide compounds for nucleic acid delivery
CN111354420B (zh) * 2020-03-08 2020-12-22 吉林大学 一种用于COVID-19病毒药物治疗的siRNA研发方法
US20220049251A1 (en) * 2020-05-20 2022-02-17 Nitto Denko Corporation dsRNA Directed to Coronavirus Proteins
CA3190097A1 (en) * 2020-08-04 2022-02-10 Tuojie Biotech (Shanghai) Co., Ltd. Modified sirna with reduced off-target activity

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BR112018008344A2 (pt) * 2015-12-13 2018-10-30 Nitto Denko Corp molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição

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Publication number Publication date
RU2018125593A3 (pt) 2020-04-06
EP3386519B1 (en) 2021-03-24
ES2865030T3 (es) 2021-10-14
US11926831B2 (en) 2024-03-12
KR20180086260A (ko) 2018-07-30
CN108367022A (zh) 2018-08-03
EP3816287A1 (en) 2021-05-05
US20190323015A1 (en) 2019-10-24
TW201726920A (zh) 2017-08-01
PT3386519T (pt) 2021-04-27
WO2017106111A1 (en) 2017-06-22
EP3386519A4 (en) 2018-11-07
US11390871B2 (en) 2022-07-19
RU2018125593A (ru) 2020-01-13
JP2018537104A (ja) 2018-12-20
US20220267779A1 (en) 2022-08-25
JP6457704B2 (ja) 2019-01-23
US10358647B2 (en) 2019-07-23
AU2016371624B2 (en) 2020-08-27
AU2016371624A1 (en) 2018-07-12
TWI752927B (zh) 2022-01-21
US20170166898A1 (en) 2017-06-15
CA3005937C (en) 2021-11-09
CA3005937A1 (en) 2017-06-22
EP3386519A1 (en) 2018-10-17

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]