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
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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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    • 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
    • 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]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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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
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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
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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 ===---===
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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
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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
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    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

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  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
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  • 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) 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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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
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
AU2019274461B2 (en) 2025-07-31
BR112020023436A2 (pt) 2021-02-23
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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