MX2022015169A - Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos. - Google Patents

Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.

Info

Publication number
MX2022015169A
MX2022015169A MX2022015169A MX2022015169A MX2022015169A MX 2022015169 A MX2022015169 A MX 2022015169A MX 2022015169 A MX2022015169 A MX 2022015169A MX 2022015169 A MX2022015169 A MX 2022015169A MX 2022015169 A MX2022015169 A MX 2022015169A
Authority
MX
Mexico
Prior art keywords
methods
rnai constructs
hsd17b13 expression
inhibiting
expression
Prior art date
Application number
MX2022015169A
Other languages
English (en)
Inventor
Justin K Murray
Bradley J Herberich
Michael Ollmann
Oliver Homann
Daniel C H Lin
Amrita Das
Patrick Collins
Original Assignee
Amgen 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 Amgen Inc filed Critical Amgen Inc
Publication of MX2022015169A publication Critical patent/MX2022015169A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3523Allyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a constructos de ARNi para reducir la expresión del gen de HSD17B13. También se describen métodos de uso de tales constructos de ARNi para tratar o prevenir enfermedad hepática, enfermedad por hígado graso no alcohólico (NAFLD).
MX2022015169A 2020-06-01 2021-06-03 Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos. MX2022015169A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063033128P 2020-06-01 2020-06-01
PCT/US2021/035730 WO2021247885A2 (en) 2020-06-01 2021-06-03 Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2022015169A true MX2022015169A (es) 2023-07-03

Family

ID=76921296

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022015169A MX2022015169A (es) 2020-06-01 2021-06-03 Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.

Country Status (12)

Country Link
US (1) US20230279399A1 (es)
EP (1) EP4158026A2 (es)
JP (1) JP2023528608A (es)
KR (1) KR20240004092A (es)
CN (1) CN116234907A (es)
AU (1) AU2021284377A1 (es)
BR (1) BR112022024501A2 (es)
CA (1) CA3184345A1 (es)
CL (1) CL2022003369A1 (es)
IL (1) IL298697A (es)
MX (1) MX2022015169A (es)
WO (1) WO2021247885A2 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202340466A (zh) * 2021-12-16 2023-10-16 大陸商上海拓界生物醫藥科技有限公司 一種dsrna、其製備方法及應用
CN116515835A (zh) * 2022-04-29 2023-08-01 北京福元医药股份有限公司 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途
WO2023213284A1 (zh) * 2022-05-06 2023-11-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2024023256A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024059165A1 (en) * 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
DE69233087T2 (de) 1991-11-22 2003-12-24 Affymetrix Inc N D Ges D Staat Verfahren zur Herstellung von Polymerarrays
US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
ES2231819T3 (es) 1995-06-07 2005-05-16 Inex Pharmaceuticals Corp Particulas de lipido-acido nucleico preparadas a traves de un intermedio complejo de lipido-acido nucleico hidrofobo y uso para transferir genes.
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
CA2294988C (en) 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
DE69906977T2 (de) 1998-07-20 2004-05-19 Protiva Biotherapeutics Inc., Burnaby In liposomen verkapselte nukleinsäurekomplexe
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
CA2487274A1 (en) 2002-05-06 2003-11-13 Nucleonics Inc. Spermine chemically linked to lipids and cell-specific targeting molecules as a transfection agent
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
EP1620544B1 (en) 2003-04-17 2018-09-19 Alnylam Pharmaceuticals Inc. MODIFIED iRNA AGENTS
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
US20090247608A1 (en) 2007-12-04 2009-10-01 Alnylam Pharmaceuticals, Inc. Targeting Lipids
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
AU2014284152B2 (en) 2013-06-21 2020-01-23 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
CA3091146A1 (en) * 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17.beta.-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
SG11202101698WA (en) * 2018-09-19 2021-04-29 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use

Also Published As

Publication number Publication date
CN116234907A (zh) 2023-06-06
US20230279399A1 (en) 2023-09-07
AU2021284377A1 (en) 2022-12-22
BR112022024501A2 (pt) 2023-05-09
CL2022003369A1 (es) 2023-07-21
KR20240004092A (ko) 2024-01-11
IL298697A (en) 2023-02-01
WO2021247885A3 (en) 2022-02-10
CA3184345A1 (en) 2021-12-09
JP2023528608A (ja) 2023-07-05
EP4158026A2 (en) 2023-04-05
WO2021247885A2 (en) 2021-12-09

Similar Documents

Publication Publication Date Title
MX2022015169A (es) Constructos de arni para inhibir la expresion de hsd17b13 y metodos de uso de los mismos.
PH12020550833A1 (en) Rnai constructs for inhibiting pnpla3 expression
MX2021014465A (es) Construcciones de arni para inhibir la expresion de scap y metodos de uso de las mismas.
MX2021006784A (es) Constructos de arni para inhibir la expresion de pnpla3 y metodos de uso de estos.
MX2022005216A (es) Piridazinonas como inhibidores de poli(adp-ribosa) polimerasa 7 (parp7).
ZA202108348B (en) Angiotensinogen (agt) irna compositions and methods of use thereof
MX2021006745A (es) Constructos de iarn modificados quimicamente y usos de estos.
MX2019002271A (es) Construcciones de arni para inhibir expresion de asgr1 y metodos para su uso.
MX2022009596A (es) Anticuerpos anti-ror1 y composiciones.
CA3139615A1 (en) Reduced nicotinamideribosides for the treatment/prevention of liver disease
MX2022002682A (es) Anticuerpos anti-cd73.
MX2023001786A (es) Construcciones de iarn y metodos para inhibir la expresion de marc1.
WO2019126097A8 (en) High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
MX2017011298A (es) Métodos para tratar cáncer que albergan la pérdida hemicigota de tp53.
MX2022004373A (es) Celulas madre modificadas y metodos de uso de las mismas.
TN2011000459A1 (en) Vaccine compositions and methods for treatment of mucormycosis and other fungal diseases
MX2019002579A (es) Composiciones y metodos de uso de depuradores de gamma-cetoaldehido para tratar, evitar o mejorar la enfermedad del higado graso no alcoholico (nafld), nash, ald o condiciones relacionadas al higado.
WO2023069754A3 (en) Rnai constructs for inhibiting gpam expression and methods of use thereof
WO2023230495A3 (en) Rnai constructs for inhibiting scap expression and methods of use thereof
MX2022007144A (es) Composiciones de acido ribonucleico de interferencia (arni) que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus metodos de uso.
WO2020104649A3 (en) Novel rna compositions and methods for inhibiting angptl8
MX2021001393A (es) Composiciones para el tratamiento de la enfermedad de injerto contra huesped.
MX2022001864A (es) Construcciones de arni para inhibir la expresión de slc30a8 y métodos de uso de las mismas.
WO2023183801A3 (en) Rnai constructs for inhibiting pnpla3 expression and methods of use thereof
TN2010000559A1 (en) Treatment for nafld