MA53924A - Modulateurs de l'expression d'apol1 - Google Patents

Modulateurs de l'expression d'apol1

Info

Publication number
MA53924A
MA53924A MA053924A MA53924A MA53924A MA 53924 A MA53924 A MA 53924A MA 053924 A MA053924 A MA 053924A MA 53924 A MA53924 A MA 53924A MA 53924 A MA53924 A MA 53924A
Authority
MA
Morocco
Prior art keywords
expression modulators
apol1 expression
apol1
modulators
expression
Prior art date
Application number
MA053924A
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 MA53924A publication Critical patent/MA53924A/fr

Links

Classifications

    • 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
    • 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
    • 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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (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)
MA053924A 2018-05-22 2019-05-21 Modulateurs de l'expression d'apol1 MA53924A (fr)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
MA53924A true MA53924A (fr) 2021-08-25

Family

ID=68613945

Family Applications (1)

Application Number Title Priority Date Filing Date
MA053924A MA53924A (fr) 2018-05-22 2019-05-21 Modulateurs de l'expression d'apol1

Country Status (27)

Country Link
US (4) US10927377B2 (fr)
EP (1) EP3799570A4 (fr)
JP (3) JP7247227B2 (fr)
KR (2) KR20250153881A (fr)
CN (3) CN118127019A (fr)
AR (1) AR115416A1 (fr)
AU (2) AU2019274461B2 (fr)
BR (1) BR112020023436A2 (fr)
CA (1) CA3099750A1 (fr)
CL (1) CL2020003010A1 (fr)
CO (1) CO2020015377A2 (fr)
CR (1) CR20200631A (fr)
EA (1) EA202092748A1 (fr)
EC (1) ECSP20082339A (fr)
IL (2) IL316397A (fr)
JO (1) JOP20200291A1 (fr)
MA (1) MA53924A (fr)
MX (2) MX2020012497A (fr)
MY (1) MY200352A (fr)
NI (1) NI202000085A (fr)
PE (1) PE20211397A1 (fr)
PH (1) PH12020551995A1 (fr)
SG (1) SG11202011397QA (fr)
TW (2) TWI841564B (fr)
UA (1) UA128819C2 (fr)
WO (1) WO2019226611A1 (fr)
ZA (1) ZA202007537B (fr)

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
IL316397A (en) 2018-05-22 2024-12-01 Ionis Pharmaceuticals Inc Modulators of APOL1 expression
IL283592B2 (en) 2018-12-17 2025-08-01 Vertex Pharma Inhibitors of apol1 and methods of using same
EP4097083A1 (fr) 2020-01-29 2022-12-07 Vertex Pharmaceuticals Incorporated Inhibiteurs d'apol1 et leurs méthodes d'utilisation
CN116157393A (zh) 2020-06-12 2023-05-23 弗特克斯药品有限公司 Apol1的抑制剂及其用途
EP4165036A1 (fr) 2020-06-12 2023-04-19 Vertex Pharmaceuticals Incorporated Formes solides d'un inhibiteur d'apol1 et utilisation de celles-ci
KR20240149461A (ko) 2022-01-18 2024-10-14 메이즈 테라퓨틱스, 인코퍼레이티드 Apol1 저해제 및 사용 방법
EP4573077A1 (fr) * 2022-08-19 2025-06-25 Maze Therapeutics, Inc. Inhibiteurs d'apol1 et procédés d'utilisation
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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US20020082401A1 (en) 1994-12-15 2002-06-27 Yeda Research And Development Company Modulators of the function of FAS/APO1 receptors
US6833361B2 (en) * 1998-05-26 2004-12-21 Ribapharm, Inc. Nucleosides having bicyclic sugar moiety
WO2002028999A2 (fr) 2000-10-03 2002-04-11 Gene Logic, Inc. Profils d'expression genique dans les cellules granulocytaires
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
WO2006034348A2 (fr) 2004-09-17 2006-03-30 Isis Pharmaceuticals, Inc. Oligonucleotides antisens ameliores
EP1867338A1 (fr) 2006-05-30 2007-12-19 Université Libre De Bruxelles Composition pharmaceutique comprenant des apolipoprotéines
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
CA2739464C (fr) 2008-10-03 2020-03-31 Curna, Inc. Traitement de maladies liees a l'apolipoproteine a1 par l'inhibition d'un produit de la transcription antisens naturel de l'apolipoproteine a1
CN102307997B (zh) 2008-12-04 2018-03-30 库尔纳公司 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1(sirt1)相关的疾病
WO2011020865A1 (fr) 2009-08-18 2011-02-24 Universite Libre De Bruxelles Variants d’apolipoprotéine l-i et leur utilisation
JP5991922B2 (ja) 2009-11-13 2016-09-14 サレプタ セラピューティクス， インコーポレイテッド アンチセンス抗ウイルス性化合物およびインフルエンザウイルス感染を処置するための方法
JP6031356B2 (ja) 2009-12-23 2016-11-24 カッパーアールエヌエー，インコーポレイテッド Ｕｃｐ２に対する天然アンチセンス転写産物の阻害による脱共役タンパク質２（ｕｃｐ２）関連疾患の治療
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
CA2863958A1 (fr) * 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Methodes et compositions permettant de moduler l'expression du facteur vii
HUE051680T2 (hu) 2012-05-24 2021-03-29 Ionis Pharmaceuticals Inc Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények
WO2014078213A2 (fr) 2012-11-16 2014-05-22 Merck Sharp & Dohme Corp. Polypeptides apol1 modifiés
WO2014085154A1 (fr) 2012-11-27 2014-06-05 Beth Israel Deaconess Medical Center, Inc. Méthodes de traitement d'une maladie rénale
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
GB201311057D0 (en) 2013-06-21 2013-08-07 Univ Greenwich Antisense oligonucleotide compositions
TWI657819B (zh) * 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
WO2015136509A2 (fr) 2014-03-14 2015-09-17 Genesis Theranostix Korlatolt Felelossegu Tarsasag Cibles diagnostiques et thérapeutiques de la prééclampsie et d'autres complications étroitement associées à la grossesse
CN116970607A (zh) 2014-03-19 2023-10-31 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
EP3020813A1 (fr) 2014-11-16 2016-05-18 Neurovision Pharma GmbH Oligonculéotides antisens en tant qu'inhibiteurs de la signalisation de TGF-R
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
IL318703A (en) 2015-06-29 2025-03-01 Caris Science Inc Therapeutic oligonucleotides
HK1255699A1 (zh) * 2015-09-24 2019-08-23 Ionis Pharmaceuticals, Inc. Kras表达的调节剂
IL316397A (en) 2018-05-22 2024-12-01 Ionis Pharmaceuticals Inc Modulators of APOL1 expression

Also Published As

Publication number Publication date
JP2021525084A (ja) 2021-09-24
JOP20200291A1 (ar) 2020-11-15
KR102874776B1 (ko) 2025-10-24
US20210079395A1 (en) 2021-03-18
JP7247227B2 (ja) 2023-03-28
MY200352A (en) 2023-12-21
TWI841564B (zh) 2024-05-11
US11525136B2 (en) 2022-12-13
AU2025259982A1 (en) 2026-01-22
JP2025013513A (ja) 2025-01-24
CN118127019A (zh) 2024-06-04
US10927377B2 (en) 2021-02-23
CR20200631A (es) 2021-02-10
TW202430638A (zh) 2024-08-01
PE20211397A1 (es) 2021-07-27
AU2019274461B2 (en) 2025-07-31
AU2019274461A1 (en) 2021-01-14
ZA202007537B (en) 2021-09-29
IL278785B1 (en) 2024-11-01
US20230265428A1 (en) 2023-08-24
CA3099750A1 (fr) 2019-11-28
IL316397A (en) 2024-12-01
CN112423792B (zh) 2024-04-23
BR112020023436A2 (pt) 2021-02-23
MX2025003294A (es) 2025-05-02
CN118127020A (zh) 2024-06-04
US12168766B2 (en) 2024-12-17
UA128819C2 (uk) 2024-10-30
WO2019226611A1 (fr) 2019-11-28
US20250066785A1 (en) 2025-02-27
CL2020003010A1 (es) 2021-06-11
AR115416A1 (es) 2021-01-13
IL278785A (en) 2021-01-31
CN112423792A (zh) 2021-02-26
EA202092748A1 (ru) 2021-04-19
CO2020015377A2 (es) 2020-12-21
EP3799570A4 (fr) 2022-04-27
JP2023075283A (ja) 2023-05-30
EP3799570A1 (fr) 2021-04-07
MX2020012497A (es) 2021-02-15
TW202016305A (zh) 2020-05-01
KR20210011981A (ko) 2021-02-02
SG11202011397QA (en) 2020-12-30
IL278785B2 (en) 2025-03-01
KR20250153881A (ko) 2025-10-27
ECSP20082339A (es) 2021-02-26
US20190359981A1 (en) 2019-11-28
JP7645301B2 (ja) 2025-03-13
NI202000085A (es) 2021-03-11
PH12020551995A1 (en) 2021-09-06

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