PE20211397A1 - Moduladores de la expresion de apol1 - Google Patents

Moduladores de la expresion de apol1

Info

Publication number
PE20211397A1
PE20211397A1 PE2020001880A PE2020001880A PE20211397A1 PE 20211397 A1 PE20211397 A1 PE 20211397A1 PE 2020001880 A PE2020001880 A PE 2020001880A PE 2020001880 A PE2020001880 A PE 2020001880A PE 20211397 A1 PE20211397 A1 PE 20211397A1
Authority
PE
Peru
Prior art keywords
apol1
modified
modulators
seq
reducing
Prior art date
Application number
PE2020001880A
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 PE20211397A1 publication Critical patent/PE20211397A1/es

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    • 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
    • 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
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • 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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Plural Heterocyclic Compounds (AREA)

Abstract

La presente invencion se refiere a un compuesto que comprende un oligonucleotido modificado de 8 a 80 nucleosidos unidos que comprende una o cualquiera de las SEQ ID NO: 13-1941, que comprende al menos 8, al menos 9, al menos 10, al menos 11 o al menos 12 bases nitrogenadas de las SEQ ID NO: 13-1941; dicho oligonucleotido modificado comprende al menos una modificacion seleccionada entre al menos un enlace entre nucleosidos modificado, un azucar modificado o una base nitrogenada modificada, tambien se refiere a composiciones y metodos que comprenden el oligonucleotido el cual inhibe la expresion de APOL1, al reducir la cantidad o actividad del ARNm de APOL1 o reducir la cantidad de proteina de APOL1, lo que puede ser util en nefropatias asociadas a APOL1.
PE2020001880A 2018-05-22 2019-05-21 Moduladores de la expresion de apol1 PE20211397A1 (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
PE20211397A1 true PE20211397A1 (es) 2021-07-27

Family

ID=68613945

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2020001880A PE20211397A1 (es) 2018-05-22 2019-05-21 Moduladores de la expresion de apol1

Country Status (24)

Country Link
US (3) US10927377B2 (es)
EP (1) EP3799570A4 (es)
JP (2) JP7247227B2 (es)
KR (1) KR20210011981A (es)
CN (1) CN112423792B (es)
AR (1) AR115416A1 (es)
AU (1) AU2019274461A1 (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 (1) IL278785A (es)
JO (1) JOP20200291A1 (es)
MA (1) MA53924A (es)
MX (1) MX2020012497A (es)
NI (1) NI202000085A (es)
PE (1) PE20211397A1 (es)
PH (1) PH12020551995A1 (es)
SG (1) SG11202011397QA (es)
WO (1) WO2019226611A1 (es)
ZA (1) ZA202007537B (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
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
MA53924A (fr) 2018-05-22 2021-08-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression d'apol1
WO2023141432A2 (en) 2022-01-18 2023-07-27 Maze Therapeutics, Inc. Apol1 inhibitors and methods of use
WO2024040235A1 (en) * 2022-08-19 2024-02-22 Maze Therapeutics, Inc. Apol1 inhibitors and methods of use

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6833361B2 (en) * 1998-05-26 2004-12-21 Ribapharm, Inc. Nucleosides having bicyclic sugar moiety
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
US9023355B2 (en) 2010-04-13 2015-05-05 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for treating renal disease
CA2863958A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Methods and compositions for modulating factor vii expression
WO2014085154A1 (en) * 2012-11-27 2014-06-05 Beth Israel Deaconess Medical Center, Inc. Methods for treating renal disease
US20160003808A1 (en) * 2013-03-14 2016-01-07 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of diseases associated with epithelial mesenchymal transition
TW202246503A (zh) * 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
TW201718010A (zh) * 2015-06-19 2017-06-01 健臻公司 治療原發性局部節段性腎小球硬化症之方法
US20180273577A1 (en) 2015-09-24 2018-09-27 Ionis Pharmaceuticals, Inc. Modulators of kras expression
MA53924A (fr) 2018-05-22 2021-08-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression d'apol1

Also Published As

Publication number Publication date
EA202092748A1 (ru) 2021-04-19
US20230265428A1 (en) 2023-08-24
US20190359981A1 (en) 2019-11-28
US11525136B2 (en) 2022-12-13
JP7247227B2 (ja) 2023-03-28
CL2020003010A1 (es) 2021-06-11
ECSP20082339A (es) 2021-02-26
AR115416A1 (es) 2021-01-13
PH12020551995A1 (en) 2021-09-06
CA3099750A1 (en) 2019-11-28
US20210079395A1 (en) 2021-03-18
US10927377B2 (en) 2021-02-23
EP3799570A4 (en) 2022-04-27
TW202016305A (zh) 2020-05-01
CR20200631A (es) 2021-02-10
JP2021525084A (ja) 2021-09-24
CN112423792A (zh) 2021-02-26
SG11202011397QA (en) 2020-12-30
ZA202007537B (en) 2021-09-29
NI202000085A (es) 2021-03-11
CN112423792B (zh) 2024-04-23
JP2023075283A (ja) 2023-05-30
EP3799570A1 (en) 2021-04-07
JOP20200291A1 (ar) 2020-11-15
WO2019226611A1 (en) 2019-11-28
MA53924A (fr) 2021-08-25
AU2019274461A1 (en) 2021-01-14
IL278785A (en) 2021-01-31
KR20210011981A (ko) 2021-02-02
MX2020012497A (es) 2021-02-15
BR112020023436A2 (pt) 2021-02-23
CO2020015377A2 (es) 2020-12-21

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