CO2020015377A2 - Moduladores de la expresión de apol1 - Google Patents

Moduladores de la expresión de apol1

Info

Publication number
CO2020015377A2
CO2020015377A2 CONC2020/0015377A CO2020015377A CO2020015377A2 CO 2020015377 A2 CO2020015377 A2 CO 2020015377A2 CO 2020015377 A CO2020015377 A CO 2020015377A CO 2020015377 A2 CO2020015377 A2 CO 2020015377A2
Authority
CO
Colombia
Prior art keywords
modulators
apol1
expression
apol1 expression
useful
Prior art date
Application number
CONC2020/0015377A
Other languages
English (en)
Spanish (es)
Inventor
Susan M Freier
Original Assignee
Ionis Pharmaceuticals Inc
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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of CO2020015377A2 publication Critical patent/CO2020015377A2/es

Links

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    • 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
    • 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
    • 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
    • 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
    • C12N2310/323Chemical 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
    • 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
    • C12N2310/346Spatial 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
    • 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
    • C12N2310/35Nature 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)
CONC2020/0015377A 2018-05-22 2020-12-09 Moduladores de la expresión de apol1 CO2020015377A2 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
CO2020015377A2 true CO2020015377A2 (es) 2020-12-21

Family

ID=68613945

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0015377A CO2020015377A2 (es) 2018-05-22 2020-12-09 Moduladores de la expresión de apol1

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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US10130632B2 (en) * 2012-11-27 2018-11-20 Beth Israel Deaconess Medical Center, Inc. Methods for treating renal 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
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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