JP2023530072A - Ｐｍｐ２２を調節するための化合物及び方法 - Google Patents

Ｐｍｐ２２を調節するための化合物及び方法 Download PDF

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Publication number
JP2023530072A
JP2023530072A JP2022570473A JP2022570473A JP2023530072A JP 2023530072 A JP2023530072 A JP 2023530072A JP 2022570473 A JP2022570473 A JP 2022570473A JP 2022570473 A JP2022570473 A JP 2022570473A JP 2023530072 A JP2023530072 A JP 2023530072A
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modified
oligomeric compound
modified oligonucleotide
oligomeric
alkyl
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JP2022570473A
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Japanese (ja)
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JP2023530072A5 (https=
JPWO2021258011A5 (https=
Inventor
スウェイジ，エリック・イー
コルダシェヴィッチ，ホリー
セス，プニット・ピー
チャオ，ヒエン・トイ
ミガワ，マイケル・ティー
バラス，ルーベン・イー
プラカシュ，タジャ・ピー
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2023530072A publication Critical patent/JP2023530072A/ja
Publication of JP2023530072A5 publication Critical patent/JP2023530072A5/ja
Publication of JPWO2021258011A5 publication Critical patent/JPWO2021258011A5/ja
Pending legal-status Critical Current

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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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  • Animal Behavior & Ethology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
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  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
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  • Hospice & Palliative Care (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022570473A 2020-06-19 2021-06-18 Ｐｍｐ２２を調節するための化合物及び方法 Pending JP2023530072A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063041643P 2020-06-19 2020-06-19
US202063041474P 2020-06-19 2020-06-19
US63/041,474 2020-06-19
US63/041,643 2020-06-19
PCT/US2021/038126 WO2021258011A1 (en) 2020-06-19 2021-06-18 Compounds and methods for modulating pmp22

Publications (3)

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JP2023530072A true JP2023530072A (ja) 2023-07-13
JP2023530072A5 JP2023530072A5 (https=) 2024-06-24
JPWO2021258011A5 JPWO2021258011A5 (https=) 2024-06-24

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US (1) US20230374519A1 (https=)
EP (1) EP4168549A4 (https=)
JP (1) JP2023530072A (https=)
WO (1) WO2021258011A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017229778A1 (en) 2016-03-09 2018-08-16 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
WO2025150052A1 (en) * 2024-01-12 2025-07-17 Potentia Ltd. Targeted delivery of drug conjugates to schwann cells and treatment methods in schwann cell-related diseases

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017156242A1 (en) * 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2019157531A1 (en) * 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2020064749A1 (en) * 2018-09-25 2020-04-02 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
JP7561129B2 (ja) * 2018-12-21 2024-10-03 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２の発現を低減するための化合物及び方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101869570B1 (ko) * 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
JP2023501352A (ja) * 2019-11-08 2023-01-18 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｓｐｄｅｆ発現を低減するための化合物及び方法

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017156242A1 (en) * 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2019157531A1 (en) * 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2020064749A1 (en) * 2018-09-25 2020-04-02 Centre National De La Recherche Scientifique Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
JP7561129B2 (ja) * 2018-12-21 2024-10-03 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｍｐ２２の発現を低減するための化合物及び方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
NUCLEIC ACIDS RESEARCH, vol. 47, no. 12, JPN6025031976, 2019, pages 6029 - 6044, ISSN: 0005655258 *
THE JOURNAL OF CLINICAL INVESTIGATION, vol. 128, no. 1, JPN6025031975, 2018, pages 359 - 368, ISSN: 0005655257 *

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WO2021258011A1 (en) 2021-12-23
EP4168549A4 (en) 2024-10-09
EP4168549A1 (en) 2023-04-26

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