CA3099750A1 - Modulators of apol1 expression - Google Patents

Modulators of apol1 expression Download PDF

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Publication number
CA3099750A1
CA3099750A1 CA3099750A CA3099750A CA3099750A1 CA 3099750 A1 CA3099750 A1 CA 3099750A1 CA 3099750 A CA3099750 A CA 3099750A CA 3099750 A CA3099750 A CA 3099750A CA 3099750 A1 CA3099750 A1 CA 3099750A1
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Prior art keywords
compound
certain embodiments
modified
seq
oligonucleotide
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Pending
Application number
CA3099750A
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English (en)
French (fr)
Inventor
Susan M. Freier
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of CA3099750A1 publication Critical patent/CA3099750A1/en
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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
    • 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
    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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

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  • 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)
CA3099750A 2018-05-22 2019-05-21 Modulators of apol1 expression Pending CA3099750A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3099750A1 true CA3099750A1 (en) 2019-11-28

Family

ID=68613945

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3099750A Pending CA3099750A1 (en) 2018-05-22 2019-05-21 Modulators of apol1 expression

Country Status (27)

Country Link
US (4) US10927377B2 (enExample)
EP (1) EP3799570A4 (enExample)
JP (3) JP7247227B2 (enExample)
KR (2) KR20250153881A (enExample)
CN (3) CN118127019A (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) IL316397A (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) TW202430638A (enExample)
UA (1) UA128819C2 (enExample)
WO (1) WO2019226611A1 (enExample)
ZA (1) ZA202007537B (enExample)

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Publication number Priority date Publication date Assignee Title
USRE49076E1 (en) 2010-04-18 2022-05-17 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for treating renal disease
CN118127019A (zh) 2018-05-22 2024-06-04 Ionis制药公司 Apol1表达的调节剂
LT3897833T (lt) 2018-12-17 2026-04-27 Vertex Pharmaceuticals Incorporated Apol1 inhibitoriai ir jų panaudojimo būdai
EP4097083A1 (en) 2020-01-29 2022-12-07 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and methods of using same
JP2023530271A (ja) 2020-06-12 2023-07-14 バーテックス ファーマシューティカルズ インコーポレイテッド Apol1の阻害剤及びこれを使用する方法
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
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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MY188457A (en) 2008-10-03 2021-12-10 Opko Curna Llc Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
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BR112020023436A2 (pt) 2021-02-23
CO2020015377A2 (es) 2020-12-21
CN118127019A (zh) 2024-06-04
KR102874776B1 (ko) 2025-10-24
PE20211397A1 (es) 2021-07-27
JP2023075283A (ja) 2023-05-30
US20210079395A1 (en) 2021-03-18
MX2025003294A (es) 2025-05-02
IL278785A (en) 2021-01-31
US20190359981A1 (en) 2019-11-28
ECSP20082339A (es) 2021-02-26
JP2025013513A (ja) 2025-01-24
WO2019226611A1 (en) 2019-11-28
IL316397A (en) 2024-12-01
EP3799570A1 (en) 2021-04-07
MX2020012497A (es) 2021-02-15
JP7247227B2 (ja) 2023-03-28
NI202000085A (es) 2021-03-11
JOP20200291A1 (ar) 2020-11-15
MA53924A (fr) 2021-08-25
US10927377B2 (en) 2021-02-23
JP2021525084A (ja) 2021-09-24
TW202016305A (zh) 2020-05-01
CL2020003010A1 (es) 2021-06-11
CR20200631A (es) 2021-02-10
MY200352A (en) 2023-12-21
IL278785B1 (en) 2024-11-01
PH12020551995A1 (en) 2021-09-06
KR20250153881A (ko) 2025-10-27
US12168766B2 (en) 2024-12-17
JP7645301B2 (ja) 2025-03-13
AU2019274461B2 (en) 2025-07-31
SG11202011397QA (en) 2020-12-30
EP3799570A4 (en) 2022-04-27
US20230265428A1 (en) 2023-08-24
ZA202007537B (en) 2021-09-29
UA128819C2 (uk) 2024-10-30
EA202092748A1 (ru) 2021-04-19
US20250066785A1 (en) 2025-02-27
CN112423792A (zh) 2021-02-26
CN118127020A (zh) 2024-06-04
CN112423792B (zh) 2024-04-23
TWI841564B (zh) 2024-05-11
AR115416A1 (es) 2021-01-13
KR20210011981A (ko) 2021-02-02
TW202430638A (zh) 2024-08-01
US11525136B2 (en) 2022-12-13
IL278785B2 (en) 2025-03-01
AU2025259982A1 (en) 2026-01-22
AU2019274461A1 (en) 2021-01-14

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