JP7757184B2 - シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ - Google Patents

シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ

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Publication number
JP7757184B2
JP7757184B2 JP2021540927A JP2021540927A JP7757184B2 JP 7757184 B2 JP7757184 B2 JP 7757184B2 JP 2021540927 A JP2021540927 A JP 2021540927A JP 2021540927 A JP2021540927 A JP 2021540927A JP 7757184 B2 JP7757184 B2 JP 7757184B2
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pmp22
seq
sirna
antisense rna
sequence
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JP2021540927A
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Japanese (ja)
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JP2022502084A (ja
Inventor
リリアーヌ・マッサード
シャーベル・マッサード
スーザン・ブタリ
ジョルジア・マリア・ラウラ・ウルビナーティ
パトリック・クヴルール
ディディエ・デメーレ
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Centre National de la Recherche Scientifique CNRS
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Centre National de la Recherche Scientifique CNRS
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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C12N2310/10—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
    • 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
    • C12N2310/351—Conjugate
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Nanotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2021540927A 2018-09-25 2019-09-24 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ Active JP7757184B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025071796A JP2025113267A (ja) 2018-09-25 2025-04-23 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18306241.3A EP3628735A1 (en) 2018-09-25 2018-09-25 Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease
EP18306241.3 2018-09-25
PCT/EP2019/075736 WO2020064749A1 (en) 2018-09-25 2019-09-24 Antisense rna targeting pmp22 for the treatment of charcot-marie-tooth 1a disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025071796A Division JP2025113267A (ja) 2018-09-25 2025-04-23 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ

Publications (2)

Publication Number Publication Date
JP2022502084A JP2022502084A (ja) 2022-01-11
JP7757184B2 true JP7757184B2 (ja) 2025-10-21

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021540927A Active JP7757184B2 (ja) 2018-09-25 2019-09-24 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ
JP2025071796A Pending JP2025113267A (ja) 2018-09-25 2025-04-23 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ

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JP2025071796A Pending JP2025113267A (ja) 2018-09-25 2025-04-23 シャルコー・マリー・トゥース１ａ病の治療のための、ｐｍｐ２２を標的とするアンチセンスｒｎａ

Country Status (6)

Country Link
US (1) US11939577B2 (enExample)
EP (2) EP3628735A1 (enExample)
JP (2) JP7757184B2 (enExample)
AU (1) AU2019350357B2 (enExample)
CA (1) CA3112870A1 (enExample)
WO (1) WO2020064749A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021184021A1 (en) * 2020-03-13 2021-09-16 Codiak Biosciences, Inc. Extracellular vesicle-aso constructs targeting pmp22
US20230374519A1 (en) * 2020-06-19 2023-11-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating pmp22
IL303308A (en) * 2020-12-01 2023-07-01 Res Inst Nationwide Childrens Hospital Products and methods for inhibiting the expression of myelin protein kp-22
WO2023012165A1 (en) 2021-08-02 2023-02-09 Universite De Montpellier Compositions and methods for treating cmt1a or cmt1e diseases with rnai molecules targeting pmp22
US20240318179A1 (en) * 2021-10-22 2024-09-26 Murdoch University Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
CA3235392A1 (en) 2021-11-18 2023-05-25 Novartis Ag Compounds targeting pmp22 for the treatment of charcot-marie-tooth disease
WO2025242147A1 (zh) * 2024-05-21 2025-11-27 北京安龙生物医药有限公司 靶向外周髓鞘蛋白-22基因的寡核苷酸及其用途

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WO2017156242A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2017210364A1 (en) 2016-06-01 2017-12-07 Infectious Disease Research Institute Nanoalum particles containing a sizing agent
US20180066257A1 (en) 2016-09-05 2018-03-08 Samsung Life Public Welfare Foundation Pharmaceutical composition for treating charcot marie tooth disease

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FR2874016B1 (fr) 2004-06-30 2006-11-24 Centre Nat Rech Scient Cnrse Nanoparticules de derives de la gemcitabine
WO2011005786A2 (en) * 2009-07-06 2011-01-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for enhancing production of a biological product
KR20220044616A (ko) * 2011-04-08 2022-04-08 유니버시티 오브 레스터 Ｍａｓｐ-2 의존적 보체 활성화와 관련된 질병을 치료하는 방법
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WO2017156242A1 (en) 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2017210364A1 (en) 2016-06-01 2017-12-07 Infectious Disease Research Institute Nanoalum particles containing a sizing agent
US20180066257A1 (en) 2016-09-05 2018-03-08 Samsung Life Public Welfare Foundation Pharmaceutical composition for treating charcot marie tooth disease

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Also Published As

Publication number Publication date
WO2020064749A1 (en) 2020-04-02
CA3112870A1 (en) 2020-04-02
AU2019350357B2 (en) 2025-10-16
US11939577B2 (en) 2024-03-26
AU2019350357A1 (en) 2021-04-22
JP2025113267A (ja) 2025-08-01
US20220002721A1 (en) 2022-01-06
EP3628735A1 (en) 2020-04-01
EP3856906A1 (en) 2021-08-04
JP2022502084A (ja) 2022-01-11

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