KR102874776B1 - Apol1 발현의 조절인자 - Google Patents

Apol1 발현의 조절인자

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Publication number
KR102874776B1
KR102874776B1 KR1020207036583A KR20207036583A KR102874776B1 KR 102874776 B1 KR102874776 B1 KR 102874776B1 KR 1020207036583 A KR1020207036583 A KR 1020207036583A KR 20207036583 A KR20207036583 A KR 20207036583A KR 102874776 B1 KR102874776 B1 KR 102874776B1
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KR
South Korea
Prior art keywords
certain embodiments
delete delete
compound
oligonucleotide
modified
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KR1020207036583A
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English (en)
Korean (ko)
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KR20210011981A (ko
Inventor
수잔 엠. 프라이어
Original Assignee
아이오니스 파마수티컬즈, 인코포레이티드
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Priority to KR1020257034730A priority Critical patent/KR20250153881A/ko
Publication of KR20210011981A publication Critical patent/KR20210011981A/ko
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Publication of KR102874776B1 publication Critical patent/KR102874776B1/ko
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    • 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
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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
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    • 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
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    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
KR1020207036583A 2018-05-22 2019-05-21 Apol1 발현의 조절인자 Active KR102874776B1 (ko)

Priority Applications (1)

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

Applications Claiming Priority (3)

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

Related Child Applications (1)

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

Publications (2)

Publication Number Publication Date
KR20210011981A KR20210011981A (ko) 2021-02-02
KR102874776B1 true KR102874776B1 (ko) 2025-10-24

Family

ID=68613945

Family Applications (2)

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020257034730A Pending KR20250153881A (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) KR102874776B1 (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
IL278785B2 (en) 2018-05-22 2025-03-01 Ionis Pharmaceuticals Inc Modulators of apol1 expression
US11618746B2 (en) 2018-12-17 2023-04-04 Vertex Pharmaceuticals Incorporated Inhibitors of APOL1 and methods of using same
WO2021154997A1 (en) 2020-01-29 2021-08-05 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and methods of using same
CN115867543A (zh) 2020-06-12 2023-03-28 弗特克斯药品有限公司 Apol1抑制剂的固体形式及其使用方法
CA3185604A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and use of the same
MX2024008868A (es) 2022-01-18 2024-09-23 Maze Therapeutics Inc Inhibidores de la apolipoproteína l1 (apol1) y métodos de uso.
US20260055083A1 (en) * 2022-08-19 2026-02-26 Maze Therapeutics, Inc. Apol1 inhibitors and methods of use
WO2025242689A1 (en) 2024-05-21 2025-11-27 Astrazeneca Ab Methods for treating apol1-mediated kidney disease

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Also Published As

Publication number Publication date
EP3799570A1 (en) 2021-04-07
US20210079395A1 (en) 2021-03-18
ECSP20082339A (es) 2021-02-26
US10927377B2 (en) 2021-02-23
US11525136B2 (en) 2022-12-13
CN112423792A (zh) 2021-02-26
ZA202007537B (en) 2021-09-29
WO2019226611A1 (en) 2019-11-28
IL278785B1 (en) 2024-11-01
MA53924A (fr) 2021-08-25
CN118127020A (zh) 2024-06-04
US12168766B2 (en) 2024-12-17
IL278785B2 (en) 2025-03-01
CN112423792B (zh) 2024-04-23
TW202430638A (zh) 2024-08-01
JP2023075283A (ja) 2023-05-30
EP3799570A4 (en) 2022-04-27
TWI841564B (zh) 2024-05-11
MX2025003294A (es) 2025-05-02
MX2020012497A (es) 2021-02-15
CL2020003010A1 (es) 2021-06-11
US20190359981A1 (en) 2019-11-28
PE20211397A1 (es) 2021-07-27
CN118127019A (zh) 2024-06-04
AR115416A1 (es) 2021-01-13
CO2020015377A2 (es) 2020-12-21
AU2025259982A1 (en) 2026-01-22
JP2021525084A (ja) 2021-09-24
MY200352A (en) 2023-12-21
NI202000085A (es) 2021-03-11
JP7645301B2 (ja) 2025-03-13
JP2025013513A (ja) 2025-01-24
CR20200631A (es) 2021-02-10
EA202092748A1 (ru) 2021-04-19
TW202016305A (zh) 2020-05-01
US20230265428A1 (en) 2023-08-24
AU2019274461B2 (en) 2025-07-31
BR112020023436A2 (pt) 2021-02-23
CA3099750A1 (en) 2019-11-28
PH12020551995A1 (en) 2021-09-06
KR20250153881A (ko) 2025-10-27
IL278785A (en) 2021-01-31
UA128819C2 (uk) 2024-10-30
JP7247227B2 (ja) 2023-03-28
US20250066785A1 (en) 2025-02-27
JOP20200291A1 (ar) 2020-11-15
IL316397A (en) 2024-12-01
KR20210011981A (ko) 2021-02-02
SG11202011397QA (en) 2020-12-30
AU2019274461A1 (en) 2021-01-14

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