BR112021011294A2 - Moléculas inibidoras de ácido nucleico de fita dupla contendo um triloop - Google Patents

Moléculas inibidoras de ácido nucleico de fita dupla contendo um triloop Download PDF

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Publication number
BR112021011294A2
BR112021011294A2 BR112021011294-4A BR112021011294A BR112021011294A2 BR 112021011294 A2 BR112021011294 A2 BR 112021011294A2 BR 112021011294 A BR112021011294 A BR 112021011294A BR 112021011294 A2 BR112021011294 A2 BR 112021011294A2
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BR
Brazil
Prior art keywords
nucleic acid
double
stranded nucleic
acid inhibitor
nucleotides
Prior art date
Application number
BR112021011294-4A
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English (en)
Portuguese (pt)
Inventor
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 BR112021011294A2 publication Critical patent/BR112021011294A2/pt

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    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
BR112021011294-4A 2018-12-12 2019-11-13 Moléculas inibidoras de ácido nucleico de fita dupla contendo um triloop BR112021011294A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862778759P 2018-12-12 2018-12-12
US62/778,759 2018-12-12
PCT/US2019/061241 WO2020123083A1 (en) 2018-12-12 2019-11-13 Double-stranded nucleic acid inhibitor molecules containing a triloop

Publications (1)

Publication Number Publication Date
BR112021011294A2 true BR112021011294A2 (pt) 2021-08-31

Family

ID=71076672

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021011294-4A BR112021011294A2 (pt) 2018-12-12 2019-11-13 Moléculas inibidoras de ácido nucleico de fita dupla contendo um triloop

Country Status (13)

Country Link
US (1) US20220064640A1 (https=)
EP (1) EP3894560A4 (https=)
JP (2) JP7535047B2 (https=)
KR (1) KR20210127917A (https=)
CN (1) CN113454221A (https=)
AU (1) AU2019397247A1 (https=)
BR (1) BR112021011294A2 (https=)
CA (1) CA3122930A1 (https=)
CL (1) CL2021001552A1 (https=)
IL (1) IL283775A (https=)
MX (1) MX2021007001A (https=)
SG (1) SG11202106046XA (https=)
WO (1) WO2020123083A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop
AU2020268798A1 (en) * 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
CA3190481A1 (en) * 2020-08-04 2022-02-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting plp1 expression
AU2022315530A1 (en) 2021-07-20 2024-01-18 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
US20230374522A1 (en) * 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
WO2024088808A1 (en) 2022-10-24 2024-05-02 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1891216B1 (en) * 2005-05-25 2012-02-22 The University Of York Hybrid interfering rna
DK2341943T3 (en) * 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
CN102264898B (zh) * 2008-10-23 2013-10-16 国立大学法人东京大学 微小rna的功能抑制方法
EP2562257A4 (en) * 2010-04-19 2014-10-01 Riken PROCESS FOR STABILIZING FUNCTIONAL NUCLEIC ACIDS
JP6091424B2 (ja) * 2011-11-16 2017-03-08 公立大学法人大阪市立大学 ＲＮＡｉ分子の活性を抑制するための核酸分子
DK3569711T3 (da) * 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
US10821112B2 (en) * 2016-03-16 2020-11-03 Dicerna Pharmaceuticals, Inc. Compositions and methods for the treatment of a beta-catenin-associated disease or disorder
HUE059718T2 (hu) * 2016-09-02 2022-12-28 Dicerna Pharmaceuticals Inc 4'-foszfát analógok és azokat tartalmazó oligonukleotidok
IL271680B (en) * 2017-06-29 2022-09-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
JP2021520355A (ja) * 2018-04-13 2021-08-19 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 Ｔｍ上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子
WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop

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Publication number Publication date
CA3122930A1 (en) 2020-06-18
JP7748500B2 (ja) 2025-10-02
US20220064640A1 (en) 2022-03-03
JP2022521877A (ja) 2022-04-13
EP3894560A1 (en) 2021-10-20
EP3894560A4 (en) 2022-10-19
AU2019397247A1 (en) 2021-06-24
WO2020123083A1 (en) 2020-06-18
IL283775A (en) 2021-07-29
CL2021001552A1 (es) 2021-11-26
JP2024109582A (ja) 2024-08-14
JP7535047B2 (ja) 2024-08-15
SG11202106046XA (en) 2021-07-29
MX2021007001A (es) 2021-10-13
KR20210127917A (ko) 2021-10-25
CN113454221A (zh) 2021-09-28

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B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO 8.6 PUBLICADO NA RPI 2800 DE 03/09/2024.