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MX2025003294A - Moduladores de la expresion de apol1 - Google Patents

Moduladores de la expresion de apol1

Info

Publication number
MX2025003294A
MX2025003294A MX2025003294A MX2025003294A MX2025003294A MX 2025003294 A MX2025003294 A MX 2025003294A MX 2025003294 A MX2025003294 A MX 2025003294A MX 2025003294 A MX2025003294 A MX 2025003294A MX 2025003294 A MX2025003294 A MX 2025003294A
Authority
MX
Mexico
Prior art keywords
modulators
apol1
expression
apol1 expression
useful
Prior art date
Application number
MX2025003294A
Other languages
English (en)
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 MX2025003294A publication Critical patent/MX2025003294A/es

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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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Plural Heterocyclic Compounds (AREA)

Abstract

Las presentes realizaciones proporcionan métodos, compuestos y composiciones útiles para inhibir la expresión de APOL1, lo que puede ser útil para tratar, prevenir o mejorar una enfermedad asociada con APOL1.
MX2025003294A 2018-05-22 2020-11-20 Moduladores de la expresion de apol1 MX2025003294A (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
MX2025003294A true MX2025003294A (es) 2025-05-02

Family

ID=68613945

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2020012497A MX2020012497A (es) 2018-05-22 2019-05-21 Moduladores de la expresion de apol1.
MX2025003294A MX2025003294A (es) 2018-05-22 2020-11-20 Moduladores de la expresion de apol1

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MX2020012497A MX2020012497A (es) 2018-05-22 2019-05-21 Moduladores de la expresion de apol1.

Country Status (27)

Country Link
US (4) US10927377B2 (es)
EP (1) EP3799570A4 (es)
JP (3) JP7247227B2 (es)
KR (2) KR102874776B1 (es)
CN (3) CN112423792B (es)
AR (1) AR115416A1 (es)
AU (2) AU2019274461B2 (es)
BR (1) BR112020023436A2 (es)
CA (1) CA3099750A1 (es)
CL (1) CL2020003010A1 (es)
CO (1) CO2020015377A2 (es)
CR (1) CR20200631A (es)
EA (1) EA202092748A1 (es)
EC (1) ECSP20082339A (es)
IL (2) IL278785B2 (es)
JO (1) JOP20200291A1 (es)
MA (1) MA53924A (es)
MX (2) MX2020012497A (es)
MY (1) MY200352A (es)
NI (1) NI202000085A (es)
PE (1) PE20211397A1 (es)
PH (1) PH12020551995A1 (es)
SG (1) SG11202011397QA (es)
TW (2) TW202430638A (es)
UA (1) UA128819C2 (es)
WO (1) WO2019226611A1 (es)
ZA (1) ZA202007537B (es)

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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
PE20211397A1 (es) 2018-05-22 2021-07-27 Ionis Pharmaceuticals Inc Moduladores de la expresion de apol1
JOP20210129A1 (ar) 2018-12-17 2023-01-30 Vertex Pharma مثبطات apol1 وطرق استخدامها
AU2021213758A1 (en) 2020-01-29 2022-09-01 Vertex Pharmaceuticals Incorporated Inhibitors of APOL1 and methods of using same
WO2021252859A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and use of the same
IL314330A (en) 2022-01-18 2024-09-01 Maze Therapeutics Inc Apol1 inhibitors and methods of use
EP4573077A1 (en) * 2022-08-19 2025-06-25 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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JP2016522675A (ja) 2013-03-14 2016-08-04 ガラパゴス・ナムローゼ・フェンノートシャップＧａｌａｐａｇｏｓ Ｎ．Ｖ． 上皮間葉移行に関連した疾患の治療において有用な分子標的及び前記標的のインヒビター
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PE20211397A1 (es) 2018-05-22 2021-07-27 Ionis Pharmaceuticals Inc Moduladores de la expresion de apol1

Also Published As

Publication number Publication date
IL278785B2 (en) 2025-03-01
US10927377B2 (en) 2021-02-23
CL2020003010A1 (es) 2021-06-11
EP3799570A1 (en) 2021-04-07
AU2019274461B2 (en) 2025-07-31
EA202092748A1 (ru) 2021-04-19
CN112423792A (zh) 2021-02-26
US11525136B2 (en) 2022-12-13
TW202430638A (zh) 2024-08-01
CO2020015377A2 (es) 2020-12-21
KR20210011981A (ko) 2021-02-02
CN118127020A (zh) 2024-06-04
CA3099750A1 (en) 2019-11-28
US20250066785A1 (en) 2025-02-27
MA53924A (fr) 2021-08-25
US20210079395A1 (en) 2021-03-18
TWI841564B (zh) 2024-05-11
TW202016305A (zh) 2020-05-01
KR102874776B1 (ko) 2025-10-24
AU2025259982A1 (en) 2026-01-22
US12168766B2 (en) 2024-12-17
EP3799570A4 (en) 2022-04-27
BR112020023436A2 (pt) 2021-02-23
CN112423792B (zh) 2024-04-23
ECSP20082339A (es) 2021-02-26
JP2023075283A (ja) 2023-05-30
JP2025013513A (ja) 2025-01-24
CN118127019A (zh) 2024-06-04
JP7247227B2 (ja) 2023-03-28
US20190359981A1 (en) 2019-11-28
PE20211397A1 (es) 2021-07-27
US20230265428A1 (en) 2023-08-24
WO2019226611A1 (en) 2019-11-28
IL278785B1 (en) 2024-11-01
JP2021525084A (ja) 2021-09-24
SG11202011397QA (en) 2020-12-30
UA128819C2 (uk) 2024-10-30
AR115416A1 (es) 2021-01-13
CR20200631A (es) 2021-02-10
AU2019274461A1 (en) 2021-01-14
JP7645301B2 (ja) 2025-03-13
IL316397A (en) 2024-12-01
JOP20200291A1 (ar) 2020-11-15
MY200352A (en) 2023-12-21
IL278785A (en) 2021-01-31
PH12020551995A1 (en) 2021-09-06
NI202000085A (es) 2021-03-11
KR20250153881A (ko) 2025-10-27
MX2020012497A (es) 2021-02-15
ZA202007537B (en) 2021-09-29

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