BR112023021109A2 - Composições e métodos para modular a expressão de pnpla3 - Google Patents

Composições e métodos para modular a expressão de pnpla3

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Publication number
BR112023021109A2
BR112023021109A2 BR112023021109A BR112023021109A BR112023021109A2 BR 112023021109 A2 BR112023021109 A2 BR 112023021109A2 BR 112023021109 A BR112023021109 A BR 112023021109A BR 112023021109 A BR112023021109 A BR 112023021109A BR 112023021109 A2 BR112023021109 A2 BR 112023021109A2
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BR
Brazil
Prior art keywords
methods
compositions
modulate
pnpla3
expression
Prior art date
Application number
BR112023021109A
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English (en)
Inventor
Anton Turanov
Dale Brown Bob
T Dudek Henryk
Marc Abrams
Utsav Saxena
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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.)
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Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of BR112023021109A2 publication Critical patent/BR112023021109A2/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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C07H21/02—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 ribosyl as saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Liquid Crystal Substances (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

composições e métodos para modular a expressão de pnpla3. a presente invenção refere-se aos oligonucleotídeos e composições incluindo estes que são divulgados para inibir ou reduzir a expressão do gene da proteína 3 contendo o domínio da fosfolipase semelhante à patatina (pnpla3). métodos de produção e utilização dos oligonucleotídeos também são divulgados, particularmente usos relacionados ao tratamento de doenças, distúrbios e/ou condições associadas à expressão de pnpla3.
BR112023021109A 2021-04-14 2022-04-13 Composições e métodos para modular a expressão de pnpla3 BR112023021109A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163174932P 2021-04-14 2021-04-14
PCT/US2022/024657 WO2022221430A1 (en) 2021-04-14 2022-04-13 Compositions and methods for modulating pnpla3 expression

Publications (1)

Publication Number Publication Date
BR112023021109A2 true BR112023021109A2 (pt) 2023-12-12

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ID=81580082

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Application Number Title Priority Date Filing Date
BR112023021109A BR112023021109A2 (pt) 2021-04-14 2022-04-13 Composições e métodos para modular a expressão de pnpla3

Country Status (12)

Country Link
US (1) US20220364098A1 (pt)
EP (1) EP4323519A1 (pt)
JP (1) JP2024514880A (pt)
KR (1) KR20230171431A (pt)
CN (1) CN117120613A (pt)
AR (1) AR125351A1 (pt)
AU (1) AU2022258459A1 (pt)
BR (1) BR112023021109A2 (pt)
CA (1) CA3209418A1 (pt)
IL (1) IL307625A (pt)
TW (1) TW202300645A (pt)
WO (1) WO2022221430A1 (pt)

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
ATE227342T1 (de) 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
IL151928A0 (en) 2000-03-30 2003-04-10 Whitehead Biomedical Inst Rna sequence-specific mediators of rna interference
JP4095895B2 (ja) 2000-12-01 2008-06-04 マックス−プランク−ゲゼルシャフト ツール フォーデルング デル ヴィッセンシャフテン エー．ヴェー． Ｒｎａ干渉を媒介する短鎖ｒｎａ分子
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
ES2708944T3 (es) 2008-09-22 2019-04-12 Dicerna Pharmaceuticals Inc Composiciones y métodos para la inhibición específica de la expresión de genes por DSRNA que tenga modificaciones
EP3067359A1 (en) 2008-09-23 2016-09-14 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
CA3151965A1 (en) 2008-12-18 2010-07-15 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
WO2013063313A1 (en) 2011-10-25 2013-05-02 Isis Pharmaceuticals, Inc. Antisense modulation of gccr expression
AU2013356383B2 (en) 2012-12-06 2017-08-31 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
EP3077511A4 (en) * 2013-12-06 2017-07-05 Dicerna Pharmaceuticals Inc. Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna
US20170114341A1 (en) 2014-06-06 2017-04-27 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
EP3256587A2 (en) * 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
JP7249080B2 (ja) 2016-08-23 2023-03-30 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
CN110072530A (zh) 2016-09-02 2019-07-30 迪克纳制药公司 4′-磷酸酯类似物和包含其的寡核苷酸
US11597927B2 (en) * 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
AU2018294415A1 (en) * 2017-06-29 2020-01-16 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting HMGB1 expression
UY38003A (es) 2017-12-12 2019-06-28 Amgen Inc Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
JP2022511550A (ja) * 2018-12-10 2022-01-31 アムジエン・インコーポレーテツド ＰＮＰＬＡ３発現を阻害するためのＲＮＡｉコンストラクト
WO2021067744A1 (en) * 2019-10-02 2021-04-08 Dicerna Pharmaceuticals, Inc. Chemical modifications of small interfering rna with minimal fluorine content

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JP2024514880A (ja) 2024-04-03
CN117120613A (zh) 2023-11-24
IL307625A (en) 2023-12-01
AU2022258459A1 (en) 2023-09-14
CA3209418A1 (en) 2022-10-20
KR20230171431A (ko) 2023-12-20
EP4323519A1 (en) 2024-02-21
AR125351A1 (es) 2023-07-12
US20220364098A1 (en) 2022-11-17
WO2022221430A1 (en) 2022-10-20
TW202300645A (zh) 2023-01-01

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