AR115416A1 - Moduladores de la expresión de apol1 - Google Patents

Moduladores de la expresión de apol1

Info

Publication number
AR115416A1
AR115416A1 ARP190101365A ARP190101365A AR115416A1 AR 115416 A1 AR115416 A1 AR 115416A1 AR P190101365 A ARP190101365 A AR P190101365A AR P190101365 A ARP190101365 A AR P190101365A AR 115416 A1 AR115416 A1 AR 115416A1
Authority
AR
Argentina
Prior art keywords
segment
modified oligonucleotide
linked nucleosides
cet
nucleoside
Prior art date
Application number
ARP190101365A
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 AR115416A1 publication Critical patent/AR115416A1/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
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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 ===---===
    • 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)
ARP190101365A 2018-05-22 2019-05-22 Moduladores de la expresión de apol1 AR115416A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862674865P 2018-05-22 2018-05-22

Publications (1)

Publication Number Publication Date
AR115416A1 true AR115416A1 (es) 2021-01-13

Family

ID=68613945

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP190101365A AR115416A1 (es) 2018-05-22 2019-05-22 Moduladores de la expresión de apol1

Country Status (27)

Country Link
US (4) US10927377B2 (https=)
EP (1) EP3799570A4 (https=)
JP (3) JP7247227B2 (https=)
KR (2) KR20250153881A (https=)
CN (3) CN118127020A (https=)
AR (1) AR115416A1 (https=)
AU (2) AU2019274461B2 (https=)
BR (1) BR112020023436A2 (https=)
CA (1) CA3099750A1 (https=)
CL (1) CL2020003010A1 (https=)
CO (1) CO2020015377A2 (https=)
CR (1) CR20200631A (https=)
EA (1) EA202092748A1 (https=)
EC (1) ECSP20082339A (https=)
IL (2) IL278785B2 (https=)
JO (1) JOP20200291A1 (https=)
MA (1) MA53924A (https=)
MX (2) MX2020012497A (https=)
MY (1) MY200352A (https=)
NI (1) NI202000085A (https=)
PE (1) PE20211397A1 (https=)
PH (1) PH12020551995A1 (https=)
SG (1) SG11202011397QA (https=)
TW (2) TWI841564B (https=)
UA (1) UA128819C2 (https=)
WO (1) WO2019226611A1 (https=)
ZA (1) ZA202007537B (https=)

Families Citing this family (9)

* 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
TWI848031B (zh) 2018-12-17 2024-07-11 美商維泰克斯製藥公司 Apol1抑制劑及其使用方法
WO2021154997A1 (en) 2020-01-29 2021-08-05 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and methods of using same
CA3185604A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and use of the same
WO2021252863A1 (en) 2020-06-12 2021-12-16 Vertex Pharmaceuticals Incorporated Solid forms of apol1 inhibitor and using the same
IL314330A (en) 2022-01-18 2024-09-01 Maze Therapeutics Inc Apol1 inhibitors and methods of use
US20260055083A1 (en) * 2022-08-19 2026-02-26 Maze Therapeutics, Inc. Apol1 inhibitors and methods of use
TW202545540A (zh) 2024-05-21 2025-12-01 瑞典商阿斯特捷利康公司 用於治療apol1介導之腎病之方法

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US6833361B2 (en) * 1998-05-26 2004-12-21 Ribapharm, Inc. Nucleosides having bicyclic sugar moiety
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USH2191H1 (en) 2000-10-24 2007-06-05 Snp Consortium Identification and mapping of single nucleotide polymorphisms in the human genome
US20070054278A1 (en) 2003-11-18 2007-03-08 Applera Corporation Polymorphisms in nucleic acid molecules encoding human enzyme proteins, methods of detection and uses thereof
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
EP2397563A3 (en) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
EP1867338A1 (en) 2006-05-30 2007-12-19 Université Libre De Bruxelles Pharmaceutical composition comprising apolipoproteins for the treatment of human diseases
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
WO2010040112A2 (en) 2008-10-03 2010-04-08 Curna, Inc. Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
EP2370580B1 (en) 2008-12-04 2019-09-11 CuRNA, Inc. Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirtuin 1
WO2011020865A1 (en) 2009-08-18 2011-02-24 Universite Libre De Bruxelles Apolipoprotein l- i variants and their use
AU2010319314C1 (en) 2009-11-13 2016-09-01 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
RU2619185C2 (ru) 2009-12-23 2017-05-12 Курна, Инк. Лечение заболеваний, связанных с разобщающим белком 2 (ucp2), путем ингибирования природного антисмыслового транскрипта к ucp2
US9023355B2 (en) 2010-04-13 2015-05-05 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for treating renal disease
US20120003644A1 (en) 2010-06-07 2012-01-05 Rappaport Family Institute For Research In The Medical Sciences Methods and kits for determining predisposition to develop kidney diseases
HK1205189A1 (en) 2012-02-08 2015-12-11 Isis Pharmaceuticals, Inc. Methods and compositions for modulating factor vii expression
RU2748495C2 (ru) 2012-05-24 2021-05-26 Ионис Фармасьютикалз, Инк. Способы и композиции для модулирования экспрессии аполипопротеина (а)
EP2920206A4 (en) 2012-11-16 2016-07-06 Merck Sharp & Dohme MODIFIED APOL1 POLYPEPTIDE
US10130632B2 (en) * 2012-11-27 2018-11-20 Beth Israel Deaconess Medical Center, Inc. Methods for treating renal disease
EP2972381A2 (en) 2013-03-14 2016-01-20 Galapagos NV Molecular targets and compounds, and methods to identify the same, useful in the treatment of diseases associated with epithelial mesenchymal transition
GB201311057D0 (en) 2013-06-21 2013-08-07 Univ Greenwich Antisense oligonucleotide compositions
TWI772856B (zh) * 2013-07-19 2022-08-01 美商百健Ma公司 用於調節τ蛋白表現之組合物
WO2015136509A2 (en) 2014-03-14 2015-09-17 Genesis Theranostix Korlatolt Felelossegu Tarsasag Diagnostic and therapeutic targets for preeclampsia and closely related complications of pregnancy
CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
EP3020813A1 (en) 2014-11-16 2016-05-18 Neurovision Pharma GmbH Antisense-oligonucleotides as inhibitors of TGF-R signaling
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
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JP6877414B2 (ja) 2015-09-24 2021-05-26 アイオニス・ファーマシューティカルズ・インコーポレイテッドIonis Pharmaceuticals,Inc. Kras発現のモジュレーター
MA53924A (fr) 2018-05-22 2021-08-25 Ionis Pharmaceuticals Inc Modulateurs de l'expression d'apol1

Also Published As

Publication number Publication date
PE20211397A1 (es) 2021-07-27
CL2020003010A1 (es) 2021-06-11
JP2025013513A (ja) 2025-01-24
CN118127020A (zh) 2024-06-04
PH12020551995A1 (en) 2021-09-06
US11525136B2 (en) 2022-12-13
IL278785B2 (en) 2025-03-01
JOP20200291A1 (ar) 2020-11-15
TW202016305A (zh) 2020-05-01
AU2019274461A1 (en) 2021-01-14
KR20250153881A (ko) 2025-10-27
AU2019274461B2 (en) 2025-07-31
SG11202011397QA (en) 2020-12-30
BR112020023436A2 (pt) 2021-02-23
KR20210011981A (ko) 2021-02-02
CA3099750A1 (en) 2019-11-28
IL278785A (en) 2021-01-31
US20210079395A1 (en) 2021-03-18
EP3799570A1 (en) 2021-04-07
TW202430638A (zh) 2024-08-01
JP2023075283A (ja) 2023-05-30
US20190359981A1 (en) 2019-11-28
JP7247227B2 (ja) 2023-03-28
JP7645301B2 (ja) 2025-03-13
WO2019226611A1 (en) 2019-11-28
CR20200631A (es) 2021-02-10
AU2025259982A1 (en) 2026-01-22
IL316397A (en) 2024-12-01
KR102874776B1 (ko) 2025-10-24
ZA202007537B (en) 2021-09-29
IL278785B1 (en) 2024-11-01
MA53924A (fr) 2021-08-25
MX2025003294A (es) 2025-05-02
US20250066785A1 (en) 2025-02-27
MY200352A (en) 2023-12-21
TWI841564B (zh) 2024-05-11
NI202000085A (es) 2021-03-11
ECSP20082339A (es) 2021-02-26
CN112423792B (zh) 2024-04-23
US10927377B2 (en) 2021-02-23
CO2020015377A2 (es) 2020-12-21
EA202092748A1 (ru) 2021-04-19
JP2021525084A (ja) 2021-09-24
CN112423792A (zh) 2021-02-26
MX2020012497A (es) 2021-02-15
CN118127019A (zh) 2024-06-04
US20230265428A1 (en) 2023-08-24
UA128819C2 (uk) 2024-10-30
US12168766B2 (en) 2024-12-17
EP3799570A4 (en) 2022-04-27

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