BR112023023807A2 - Composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1) - Google Patents

Composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1)

Info

Publication number
BR112023023807A2
BR112023023807A2 BR112023023807A BR112023023807A BR112023023807A2 BR 112023023807 A2 BR112023023807 A2 BR 112023023807A2 BR 112023023807 A BR112023023807 A BR 112023023807A BR 112023023807 A BR112023023807 A BR 112023023807A BR 112023023807 A2 BR112023023807 A2 BR 112023023807A2
Authority
BR
Brazil
Prior art keywords
marc1
expression
compositions
inhibiting
methods
Prior art date
Application number
BR112023023807A
Other languages
English (en)
Inventor
Chengjung Lai
Henryk Dudek
Wayne Pursell Natalie
Wen Han
Geoffrey Haynes William
Zhihao Ding
Original Assignee
Dicerna Pharmaceuticals Inc
Novo Nordisk As
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, Novo Nordisk As filed Critical Dicerna Pharmaceuticals Inc
Publication of BR112023023807A2 publication Critical patent/BR112023023807A2/pt

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N2310/11—Antisense
    • 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 N.A.
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/35—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

Landscapes

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

Abstract

composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1). são fornecidos, neste documento, oligonucleotídeos que inibem a expressão de marc1. também são fornecidas composições que incluem os mesmos e seus usos, particularmente usos relacionados ao tratamento de doenças, distúrbios e/ou condições associadas à expressão de marc1.
BR112023023807A 2021-05-28 2022-05-27 Composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1) BR112023023807A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163194395P 2021-05-28 2021-05-28
EP21183860 2021-07-06
PCT/EP2022/064408 WO2022248665A1 (en) 2021-05-28 2022-05-27 Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (marc1) expression

Publications (1)

Publication Number Publication Date
BR112023023807A2 true BR112023023807A2 (pt) 2024-02-20

Family

ID=82067447

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023023807A BR112023023807A2 (pt) 2021-05-28 2022-05-27 Composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1)

Country Status (14)

Country Link
US (2) US11655473B2 (pt)
EP (1) EP4347820A1 (pt)
JP (2) JP7463621B2 (pt)
KR (1) KR102662071B1 (pt)
AR (1) AR125992A1 (pt)
AU (1) AU2022282579A1 (pt)
BR (1) BR112023023807A2 (pt)
CA (1) CA3215836A1 (pt)
CL (1) CL2023003363A1 (pt)
CO (1) CO2023016136A2 (pt)
IL (1) IL308418A (pt)
MX (1) MX2023013504A (pt)
TW (1) TWI831225B (pt)
WO (1) WO2022248665A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024192379A1 (en) * 2023-03-16 2024-09-19 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of mitochondrial amidoxime reducing component 1 (marc1), pharmaceutical compositions thereof, and methods of use

Family Cites Families (27)

* 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
JPH09502092A (ja) 1993-09-02 1997-03-04 リボザイム・ファーマシューティカルズ・インコーポレイテッド 非ヌクレオチドを含有する酵素性核酸
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
DK1309726T4 (en) 2000-03-30 2019-01-28 Whitehead Inst Biomedical Res RNA Sequence-Specific Mediators of RNA Interference
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
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
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
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
NL1026610C2 (nl) * 2004-07-08 2006-01-10 Wp Suspension B V Schokbreker met drukondersteunde verstelling.
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, 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
US8513207B2 (en) 2008-12-18 2013-08-20 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
RU2014119787A (ru) 2011-10-25 2015-12-10 Айсис Фармасьютикалс, Инк. Антисмысловая регуляция экспрессии gccr
CA2970801C (en) 2014-12-15 2024-02-13 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
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
CN110799192A (zh) * 2017-03-27 2020-02-14 加利福尼亚大学董事会 治疗癌症的组合物和方法
US10835581B2 (en) 2017-11-28 2020-11-17 University of Pittsburgh—of the Commonwealth System of Higher Education Method of treating insulin resistance
US20200241005A1 (en) 2019-01-25 2020-07-30 Viscient Biosciences, Inc. Compositions and methods for the diagnosis and treatment of diseases of the liver

Also Published As

Publication number Publication date
AR125992A1 (es) 2023-08-30
EP4347820A1 (en) 2024-04-10
MX2023013504A (es) 2024-02-23
US20230042451A1 (en) 2023-02-09
JP2024071571A (ja) 2024-05-24
IL308418A (en) 2024-01-01
CO2023016136A2 (es) 2024-02-15
JP2024508069A (ja) 2024-02-22
KR102662071B1 (ko) 2024-05-03
CL2023003363A1 (es) 2024-06-21
TW202307206A (zh) 2023-02-16
US20230340490A1 (en) 2023-10-26
AU2022282579A1 (en) 2023-11-02
KR20230130609A (ko) 2023-09-12
JP7463621B2 (ja) 2024-04-08
US11655473B2 (en) 2023-05-23
TWI831225B (zh) 2024-02-01
AU2022282579A9 (en) 2023-11-16
WO2022248665A1 (en) 2022-12-01
CA3215836A1 (en) 2022-12-01

Similar Documents

Publication Publication Date Title
BR112023001957A2 (pt) Composições e métodos para inibir expressão de lpa
BR112022018667A2 (pt) Composições e métodos para inibir a expressão de ang-ptl3
BR112022016382A2 (pt) Triptaminas específicas para o uso no tratamento de doenças do humor
BR112019016497A2 (pt) 2-heteroaril-3-oxo-2,3-di-hidropiridazina-4-carboxamidas para o tratamento de câncer.
BR112018074185A2 (pt) 3-oxo-2,6-difenil-2,3-di-hidropiridazina-4-carboxamidas
BR112023017367A2 (pt) Composições e métodos para inibição de ceto-hexoquinase (khk)
ECSP21080535A (es) Compuestos y métodos para el tratamiento de covid-19
BR112018072545A2 (pt) inibidores da arginase e suas aplicações terapêuticas
BR112018075992A2 (pt) piridinas substituídas como inibidores da dnmt1
BR112021022758A2 (pt) Compostos de 1-oxo-isoindolina-5-carboxamida substituídos, suas composições e métodos de tratamento com os mesmos
BR112022012637A2 (pt) Composto de piridazinil-tiazolcarboxamida, composição farmacêutica e inibidor da dgk ¿ que compreendem o mesmo e uso do dito composto para tratar câncer relacionado à ativação das células imunes ou câncer tendo resistência à terapia com anticorpo anti-pd-1 / anticorpo anti-pd-l1
BR112017012327A2 (pt) benzamidas substituídas por 1,3-tiazol-2-il.
BR112017026739A2 (pt) compostos para uso no tratamento de distúrbios neuromusculares
BR112022019991A2 (pt) Compostos, composição farmacêutica compreendendo o mesmo, e usos dos mesmos
BR112021018739A8 (pt) Composições e métodos para o tratamento de doenças ou distúrbios associados a kras
BR112022015110A2 (pt) Compostos e usos dos mesmos
BR112018013084A2 (pt) tratamentos de combinação compreendendo a administração de imidazopirazinonas
BR112022009409A2 (pt) Novos derivados que têm grupamento 2,3-dihidro-1h-indeno ou 2,3-dihidrobenzofurano ou sal farmaceuticamente aceitável dos mesmos e composições farmacêuticas que os compreendem
BR112022018929A2 (pt) Inibidores da ciclofilina e seus usos
ECSP21088111A (es) Analogos de 3–(5–metil–1,3–tiazol–2–il)–n–{(1r)–1–[2–(trifluor–metil)pirimidin–5–il]etil}benzamida
BR112022001628A2 (pt) [1,2,4]triazolo[1,5-c]quinazolin-5-aminas
BR112018076485A2 (pt) carotenoides microbianos afetando a composição microbiana do intestino
BR112023023807A2 (pt) Composições e métodos para inibir a expressão do componente redutor da amidoxima mitocondrial 1 (marc1)
BR112022010411A2 (pt) Composições bacterianas projetadas e usos das mesmas
BR112022023983A2 (pt) Compostos terapêuticos