AU2019274461B2 - Modulators of APOL1 expression - Google Patents

Modulators of APOL1 expression

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Publication number
AU2019274461B2
AU2019274461B2 AU2019274461A AU2019274461A AU2019274461B2 AU 2019274461 B2 AU2019274461 B2 AU 2019274461B2 AU 2019274461 A AU2019274461 A AU 2019274461A AU 2019274461 A AU2019274461 A AU 2019274461A AU 2019274461 B2 AU2019274461 B2 AU 2019274461B2
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AU
Australia
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.)
Active
Application number
AU2019274461A
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English (en)
Other versions
AU2019274461A1 (en
Inventor
Susan M. Freier
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of AU2019274461A1 publication Critical patent/AU2019274461A1/en
Application granted granted Critical
Publication of AU2019274461B2 publication Critical patent/AU2019274461B2/en
Priority to AU2025259982A priority Critical patent/AU2025259982A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • 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
    • C12N2310/30—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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special 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)
AU2019274461A 2018-05-22 2019-05-21 Modulators of APOL1 expression Active AU2019274461B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025259982A AU2025259982A1 (en) 2018-05-22 2025-10-31 Modulators of apol1 expression

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
AU2025259982A Division AU2025259982A1 (en) 2018-05-22 2025-10-31 Modulators of apol1 expression

Publications (2)

Publication Number Publication Date
AU2019274461A1 AU2019274461A1 (en) 2021-01-14
AU2019274461B2 true AU2019274461B2 (en) 2025-07-31

Family

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AU2019274461A Active AU2019274461B2 (en) 2018-05-22 2019-05-21 Modulators of APOL1 expression
AU2025259982A Pending AU2025259982A1 (en) 2018-05-22 2025-10-31 Modulators of apol1 expression

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EP (1) EP3799570A4 (enExample)
JP (3) JP7247227B2 (enExample)
KR (2) KR102874776B1 (enExample)
CN (3) CN118127020A (enExample)
AR (1) AR115416A1 (enExample)
AU (2) AU2019274461B2 (enExample)
BR (1) BR112020023436A2 (enExample)
CA (1) CA3099750A1 (enExample)
CL (1) CL2020003010A1 (enExample)
CO (1) CO2020015377A2 (enExample)
CR (1) CR20200631A (enExample)
EA (1) EA202092748A1 (enExample)
EC (1) ECSP20082339A (enExample)
IL (2) IL278785B2 (enExample)
JO (1) JOP20200291A1 (enExample)
MA (1) MA53924A (enExample)
MX (2) MX2020012497A (enExample)
MY (1) MY200352A (enExample)
NI (1) NI202000085A (enExample)
PE (1) PE20211397A1 (enExample)
PH (1) PH12020551995A1 (enExample)
SG (1) SG11202011397QA (enExample)
TW (2) TWI841564B (enExample)
UA (1) UA128819C2 (enExample)
WO (1) WO2019226611A1 (enExample)
ZA (1) ZA202007537B (enExample)

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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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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
KR102874776B1 (ko) 2025-10-24
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
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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