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)
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 PE20211397A1 publication Critical patent/PE20211397A1/es

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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)
  • 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)
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 (27)

Country Link
US (4) US10927377B2 (enExample)
EP (1) EP3799570A4 (enExample)
JP (3) JP7247227B2 (enExample)
KR (2) KR20250153881A (enExample)
CN (3) CN118127019A (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) IL316397A (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)

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 (en) 2020-01-29 2022-12-07 Vertex Pharmaceuticals Incorporated Inhibitors of apol1 and methods of using same
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 (en) 2020-06-12 2023-04-19 Vertex Pharmaceuticals Incorporated Solid forms of apol1 inhibitor and using the same
KR20240149461A (ko) 2022-01-18 2024-10-14 메이즈 테라퓨틱스, 인코퍼레이티드 Apol1 저해제 및 사용 방법
EP4573077A1 (en) * 2022-08-19 2025-06-25 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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WO2002028999A2 (en) 2000-10-03 2002-04-11 Gene Logic, Inc. Gene expression profiles in granulocytic cells
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US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
WO2006034348A2 (en) 2004-09-17 2006-03-30 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
CA2739464C (en) 2008-10-03 2020-03-31 Curna, Inc. Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-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 (en) 2009-08-18 2011-02-24 Universite Libre De Bruxelles Apolipoprotein l- i variants and their use
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
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HUE051680T2 (hu) 2012-05-24 2021-03-29 Ionis Pharmaceuticals Inc Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények
WO2014078213A2 (en) 2012-11-16 2014-05-22 Merck Sharp & Dohme Corp. Modified apol1 polypeptides
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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
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 (en) 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 (en) 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
MA53924A (fr) 2021-08-25
EA202092748A1 (ru) 2021-04-19
CO2020015377A2 (es) 2020-12-21
EP3799570A4 (en) 2022-04-27
JP2023075283A (ja) 2023-05-30
EP3799570A1 (en) 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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