UY38539A - AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE HIF-2 ALFA (EPAS1), COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS - Google Patents

AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE HIF-2 ALFA (EPAS1), COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS

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Publication number
UY38539A
UY38539A UY0001038539A UY38539A UY38539A UY 38539 A UY38539 A UY 38539A UY 0001038539 A UY0001038539 A UY 0001038539A UY 38539 A UY38539 A UY 38539A UY 38539 A UY38539 A UY 38539A
Authority
UY
Uruguay
Prior art keywords
alpha
rnai agents
hif
epas1
expression
Prior art date
Application number
UY0001038539A
Other languages
English (en)
Inventor
Zhen Li
Rui Zhu
Anthony Nicholas
Erich Altenhofer
So Wong
Dongxu Shu
Jeffrey Carlson
Xiaokai Li
Matthew-Fowler-Watters
Bo Chen
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of UY38539A publication Critical patent/UY38539A/es

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    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • 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
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/30—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members
    • C07D207/34—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D207/36—Oxygen or sulfur atoms
    • C07D207/40—2,5-Pyrrolidine-diones
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    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
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    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0331—Animal model for proliferative diseases
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    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Agentes de ARNi, por ejemplo, agentes de ARNi de hebra doble, capaces de inhibir la expresión del gen de HIF-2 alfa (EPAS1). También se describen composiciones que incluyen agentes de ARNi contra HIF-2 alfa y métodos de uso de los mismos. Los agentes de ARNi contra HIF-2 alfa que se divulgan en la presente pueden estar unidos o conjugados a ligandos de direccionamiento (tales como compuestos con afinidad por las integrinas, incluyendo a las integrinas alfa-v-beta 3 y alfa-v-beta 5) y potenciadores farmacocinéticos (PK), para facilitar la administración a las células, incluyendo a las células de carcinoma renal.
UY0001038539A 2019-01-09 2020-01-09 AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE HIF-2 ALFA (EPAS1), COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS UY38539A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962790360P 2019-01-09 2019-01-09
US201962827564P 2019-04-01 2019-04-01
US201962839381P 2019-04-26 2019-04-26

Publications (1)

Publication Number Publication Date
UY38539A true UY38539A (es) 2020-07-31

Family

ID=71520182

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001038539A UY38539A (es) 2019-01-09 2020-01-09 AGENTES DE ARNi PARA INHIBIR LA EXPRESIÓN DE HIF-2 ALFA (EPAS1), COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS

Country Status (14)

Country Link
US (1) US20220204976A1 (es)
EP (1) EP3908288A4 (es)
JP (1) JP2022518384A (es)
KR (1) KR20210113273A (es)
CN (1) CN113613661A (es)
AU (1) AU2020207050A1 (es)
BR (1) BR112021013362A2 (es)
CA (1) CA3120580A1 (es)
IL (1) IL284696A (es)
MX (1) MX2021008261A (es)
SG (1) SG11202105577TA (es)
TW (1) TW202043470A (es)
UY (1) UY38539A (es)
WO (1) WO2020146521A2 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114053426A (zh) * 2020-07-30 2022-02-18 成都科岭源医药技术有限公司 一种双药链接组装单元及双药靶向接头-药物偶联物
JP2023541427A (ja) * 2020-09-11 2023-10-02 アローヘッド ファーマシューティカルズ インコーポレイテッド 骨格筋送達プラットフォーム及び使用方法
WO2022086882A1 (en) 2020-10-21 2022-04-28 Nikang Therapeutics, Inc. Diagnostic and therapeutic methods for treatment of cancer with a hif-2(alpha) inhibitor
CN115105607A (zh) * 2021-03-22 2022-09-27 成都科岭源医药技术有限公司 一种用于adc的双药-接头的制备方法及其用途
CN113304274A (zh) * 2021-04-23 2021-08-27 南开大学 缀合物及其制备方法和用途

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104225613A (zh) * 2008-03-14 2014-12-24 Visen医药公司 整联蛋白靶向试剂及使用其的体内和体外成像方法
CN102037123A (zh) * 2008-04-04 2011-04-27 卡兰多制药股份有限公司 Epas1抑制剂的组合物和用途
WO2014134255A2 (en) * 2013-02-28 2014-09-04 Novartis Ag Organic compositions to treat epas1-related diseases
KR20180011198A (ko) * 2015-05-29 2018-01-31 애로우헤드 파마슈티컬스 인코포레이티드 ＨＩＦ２α의 유전자 발현을 억제하는 조성물 및 방법

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Publication number Publication date
SG11202105577TA (en) 2021-06-29
IL284696A (en) 2021-08-31
CN113613661A (zh) 2021-11-05
WO2020146521A3 (en) 2020-08-20
US20220204976A1 (en) 2022-06-30
MX2021008261A (es) 2021-08-16
TW202043470A (zh) 2020-12-01
EP3908288A2 (en) 2021-11-17
EP3908288A4 (en) 2023-12-20
JP2022518384A (ja) 2022-03-15
BR112021013362A2 (pt) 2021-12-14
CA3120580A1 (en) 2020-07-16
KR20210113273A (ko) 2021-09-15
WO2020146521A2 (en) 2020-07-16
AU2020207050A1 (en) 2021-06-17

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