JP2023501352A - Ｓｐｄｅｆ発現を低減するための化合物及び方法 - Google Patents

Ｓｐｄｅｆ発現を低減するための化合物及び方法 Download PDF

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Publication number
JP2023501352A
JP2023501352A JP2022526072A JP2022526072A JP2023501352A JP 2023501352 A JP2023501352 A JP 2023501352A JP 2022526072 A JP2022526072 A JP 2022526072A JP 2022526072 A JP2022526072 A JP 2022526072A JP 2023501352 A JP2023501352 A JP 2023501352A
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modified
certain embodiments
oligonucleotide
oligomeric
oligomeric compound
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Japanese (ja)
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JP2023501352A5 (enExample
Inventor
フレアー，スーザン・エム
ブイ，フィン・ホア
グオ，シュリン
クロスビー，ジェフリー・アール
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pulmonology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Inorganic Chemistry (AREA)
  • Otolaryngology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022526072A 2019-11-08 2020-11-06 Ｓｐｄｅｆ発現を低減するための化合物及び方法 Pending JP2023501352A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962932758P 2019-11-08 2019-11-08
US62/932,758 2019-11-08
US202063056969P 2020-07-27 2020-07-27
US63/056,969 2020-07-27
PCT/US2020/059506 WO2021092459A1 (en) 2019-11-08 2020-11-06 Compounds and methods for reducing spdef expression

Publications (2)

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JP2023501352A true JP2023501352A (ja) 2023-01-18
JP2023501352A5 JP2023501352A5 (enExample) 2023-11-14

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JP2022526072A Pending JP2023501352A (ja) 2019-11-08 2020-11-06 Ｓｐｄｅｆ発現を低減するための化合物及び方法

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US (2) US20210139906A1 (enExample)
EP (1) EP4054655A4 (enExample)
JP (1) JP2023501352A (enExample)
TW (1) TW202128999A (enExample)
WO (1) WO2021092459A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4168549A4 (en) * 2020-06-19 2024-10-09 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR PMP22 MODULATION

Citations (6)

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JP2002521048A (ja) * 1998-07-31 2002-07-16 ヒューマン ジノーム サイエンシーズ， インコーポレイテッド 前立腺由来ｅｔｓ因子
US20090202595A1 (en) * 2007-12-21 2009-08-13 Ashwani Sood Anti-pdef antibodies and uses thereof
JP2011502515A (ja) * 2007-11-09 2011-01-27 アイシス ファーマシューティカルズ インコーポレイティッド 第９因子発現の調節
US20120053112A1 (en) * 2009-05-05 2012-03-01 Children's Hospital Medical Center Methods and compositions related to the regulation of goblet cell differentiation, mucus production and mucus secretion
US20170343551A1 (en) * 2016-04-05 2017-11-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Method of diagnosing and treating prostate cancer
WO2019169219A1 (en) * 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Modulators of irf4 expression

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US6821724B1 (en) * 1998-09-17 2004-11-23 Affymetrix, Inc. Methods of genetic analysis using nucleic acid arrays
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US20060257851A1 (en) * 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US20050221354A1 (en) * 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes
US20070048756A1 (en) * 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
AU2007225238A1 (en) * 2006-03-10 2007-09-20 Wyeth Microarray for monitoring gene expression in multiple strains of Streptococcus pneumoniae
US20090123928A1 (en) * 2007-10-11 2009-05-14 The Johns Hopkins University Genomic Landscapes of Human Breast and Colorectal Cancers
WO2010115278A1 (en) * 2009-04-06 2010-10-14 University Of Saskatchewan Methods and compositions for treating and preventing shiga toxin-producing escherichia coli infection
US20140004565A1 (en) * 2009-07-06 2014-01-02 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
US9920317B2 (en) * 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2817256A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
AR092982A1 (es) * 2012-10-11 2015-05-13 Isis Pharmaceuticals Inc Modulacion de la expresion de receptores androgenicos
US9988626B2 (en) * 2013-07-29 2018-06-05 Universität Zu Köln Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof
CA2954791C (en) * 2014-07-14 2025-11-18 The Regents Of The University Of California CRISPR/CAS TRANSCRIPTIONAL MODULATION
US20180305689A1 (en) * 2015-04-22 2018-10-25 Mina Therapeutics Limited Sarna compositions and methods of use

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JP2002521048A (ja) * 1998-07-31 2002-07-16 ヒューマン ジノーム サイエンシーズ， インコーポレイテッド 前立腺由来ｅｔｓ因子
JP2011502515A (ja) * 2007-11-09 2011-01-27 アイシス ファーマシューティカルズ インコーポレイティッド 第９因子発現の調節
US20090202595A1 (en) * 2007-12-21 2009-08-13 Ashwani Sood Anti-pdef antibodies and uses thereof
US20120053112A1 (en) * 2009-05-05 2012-03-01 Children's Hospital Medical Center Methods and compositions related to the regulation of goblet cell differentiation, mucus production and mucus secretion
US20170343551A1 (en) * 2016-04-05 2017-11-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Method of diagnosing and treating prostate cancer
WO2019169219A1 (en) * 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Modulators of irf4 expression

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EP4054655A4 (en) 2024-02-28
WO2021092459A1 (en) 2021-05-14
TW202128999A (zh) 2021-08-01
US20220290137A1 (en) 2022-09-15
EP4054655A1 (en) 2022-09-14
US20210139906A1 (en) 2021-05-13

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