KR20250153881A - Apol1 발현의 조절인자 - Google Patents

Apol1 발현의 조절인자

Info

Publication number
KR20250153881A
KR20250153881A KR1020257034730A KR20257034730A KR20250153881A KR 20250153881 A KR20250153881 A KR 20250153881A KR 1020257034730 A KR1020257034730 A KR 1020257034730A KR 20257034730 A KR20257034730 A KR 20257034730A KR 20250153881 A KR20250153881 A KR 20250153881A
Authority
KR
South Korea
Prior art keywords
certain embodiments
compound
modified
oligonucleotide
apol1
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
KR1020257034730A
Other languages
English (en)
Korean (ko)
Inventor
수잔 엠. 프라이어
Original Assignee
아이오니스 파마수티컬즈, 인코포레이티드
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 아이오니스 파마수티컬즈, 인코포레이티드 filed Critical 아이오니스 파마수티컬즈, 인코포레이티드
Publication of KR20250153881A publication Critical patent/KR20250153881A/ko
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • 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
    • C12N2310/30Chemical 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
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    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020257034730A 2018-05-22 2019-05-21 Apol1 발현의 조절인자 Pending KR20250153881A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862674865P 2018-05-22 2018-05-22
US62/674,865 2018-05-22
KR1020207036583A KR102874776B1 (ko) 2018-05-22 2019-05-21 Apol1 발현의 조절인자
PCT/US2019/033244 WO2019226611A1 (en) 2018-05-22 2019-05-21 Modulators of apol1 expression

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020207036583A Division KR102874776B1 (ko) 2018-05-22 2019-05-21 Apol1 발현의 조절인자

Publications (1)

Publication Number Publication Date
KR20250153881A true KR20250153881A (ko) 2025-10-27

Family

ID=68613945

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020257034730A Pending KR20250153881A (ko) 2018-05-22 2019-05-21 Apol1 발현의 조절인자
KR1020207036583A Active KR102874776B1 (ko) 2018-05-22 2019-05-21 Apol1 발현의 조절인자

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020207036583A Active KR102874776B1 (ko) 2018-05-22 2019-05-21 Apol1 발현의 조절인자

Country Status (27)

Country Link
US (4) US10927377B2 (https=)
EP (1) EP3799570A4 (https=)
JP (3) JP7247227B2 (https=)
KR (2) KR20250153881A (https=)
CN (3) CN118127020A (https=)
AR (1) AR115416A1 (https=)
AU (2) AU2019274461B2 (https=)
BR (1) BR112020023436A2 (https=)
CA (1) CA3099750A1 (https=)
CL (1) CL2020003010A1 (https=)
CO (1) CO2020015377A2 (https=)
CR (1) CR20200631A (https=)
EA (1) EA202092748A1 (https=)
EC (1) ECSP20082339A (https=)
IL (2) IL278785B2 (https=)
JO (1) JOP20200291A1 (https=)
MA (1) MA53924A (https=)
MX (2) MX2020012497A (https=)
MY (1) MY200352A (https=)
NI (1) NI202000085A (https=)
PE (1) PE20211397A1 (https=)
PH (1) PH12020551995A1 (https=)
SG (1) SG11202011397QA (https=)
TW (2) TWI841564B (https=)
UA (1) UA128819C2 (https=)
WO (1) WO2019226611A1 (https=)
ZA (1) ZA202007537B (https=)

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USRE49076E1 (en) 2010-04-18 2022-05-17 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for treating renal disease
MA53924A (fr) 2018-05-22 2021-08-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression d'apol1
TWI848031B (zh) 2018-12-17 2024-07-11 美商維泰克斯製藥公司 Apol1抑制劑及其使用方法
WO2021154997A1 (en) 2020-01-29 2021-08-05 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and methods of using same
CA3185604A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and use of the same
WO2021252863A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Solid forms of apol1 inhibitor and using the same
IL314330A (en) 2022-01-18 2024-09-01 Maze Therapeutics Inc Apol1 inhibitors and methods of use
US20260055083A1 (en) * 2022-08-19 2026-02-26 Maze Therapeutics, Inc. Apol1 inhibitors and methods of use
TW202545540A (zh) 2024-05-21 2025-12-01 瑞典商阿斯特捷利康公司 用於治療apol1介導之腎病之方法

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TWI772856B (zh) * 2013-07-19 2022-08-01 美商百健Ma公司 用於調節τ蛋白表現之組合物
WO2015136509A2 (en) 2014-03-14 2015-09-17 Genesis Theranostix Korlatolt Felelossegu Tarsasag Diagnostic and therapeutic targets for preeclampsia and closely related complications of pregnancy
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EP3020813A1 (en) 2014-11-16 2016-05-18 Neurovision Pharma GmbH Antisense-oligonucleotides as inhibitors of TGF-R signaling
TW201718010A (zh) 2015-06-19 2017-06-01 健臻公司 治療原發性局部節段性腎小球硬化症之方法
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JP6877414B2 (ja) 2015-09-24 2021-05-26 アイオニス・ファーマシューティカルズ・インコーポレイテッドIonis Pharmaceuticals,Inc. Kras発現のモジュレーター
MA53924A (fr) 2018-05-22 2021-08-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression d'apol1

Also Published As

Publication number Publication date
PE20211397A1 (es) 2021-07-27
CL2020003010A1 (es) 2021-06-11
JP2025013513A (ja) 2025-01-24
CN118127020A (zh) 2024-06-04
PH12020551995A1 (en) 2021-09-06
US11525136B2 (en) 2022-12-13
IL278785B2 (en) 2025-03-01
JOP20200291A1 (ar) 2020-11-15
TW202016305A (zh) 2020-05-01
AU2019274461A1 (en) 2021-01-14
AR115416A1 (es) 2021-01-13
AU2019274461B2 (en) 2025-07-31
SG11202011397QA (en) 2020-12-30
BR112020023436A2 (pt) 2021-02-23
KR20210011981A (ko) 2021-02-02
CA3099750A1 (en) 2019-11-28
IL278785A (en) 2021-01-31
US20210079395A1 (en) 2021-03-18
EP3799570A1 (en) 2021-04-07
TW202430638A (zh) 2024-08-01
JP2023075283A (ja) 2023-05-30
US20190359981A1 (en) 2019-11-28
JP7247227B2 (ja) 2023-03-28
JP7645301B2 (ja) 2025-03-13
WO2019226611A1 (en) 2019-11-28
CR20200631A (es) 2021-02-10
AU2025259982A1 (en) 2026-01-22
IL316397A (en) 2024-12-01
KR102874776B1 (ko) 2025-10-24
ZA202007537B (en) 2021-09-29
IL278785B1 (en) 2024-11-01
MA53924A (fr) 2021-08-25
MX2025003294A (es) 2025-05-02
US20250066785A1 (en) 2025-02-27
MY200352A (en) 2023-12-21
TWI841564B (zh) 2024-05-11
NI202000085A (es) 2021-03-11
ECSP20082339A (es) 2021-02-26
CN112423792B (zh) 2024-04-23
US10927377B2 (en) 2021-02-23
CO2020015377A2 (es) 2020-12-21
EA202092748A1 (ru) 2021-04-19
JP2021525084A (ja) 2021-09-24
CN112423792A (zh) 2021-02-26
MX2020012497A (es) 2021-02-15
CN118127019A (zh) 2024-06-04
US20230265428A1 (en) 2023-08-24
UA128819C2 (uk) 2024-10-30
US12168766B2 (en) 2024-12-17
EP3799570A4 (en) 2022-04-27

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