JP2025508959A - Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 - Google Patents

Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 Download PDF

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JP2025508959A
JP2025508959A JP2024552157A JP2024552157A JP2025508959A JP 2025508959 A JP2025508959 A JP 2025508959A JP 2024552157 A JP2024552157 A JP 2024552157A JP 2024552157 A JP2024552157 A JP 2024552157A JP 2025508959 A JP2025508959 A JP 2025508959A
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oligonucleotide
pharma
acceptable salt
ceutically acceptable
seq
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JP2025508959A5 (https=
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アントニエヴィッチ、イリーナ
レイ、ダブリュー．ジョージ
グー、ペイ
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Takeda Pharmaceuticals USA Inc
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Takeda Pharmaceuticals USA Inc
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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
    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
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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
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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
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    • 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
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024552157A 2022-03-02 2023-03-01 Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 Pending JP2025508959A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263315707P 2022-03-02 2022-03-02
US63/315,707 2022-03-02
PCT/US2023/063531 WO2023168304A2 (en) 2022-03-02 2023-03-01 Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity

Publications (2)

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JP2025508959A true JP2025508959A (ja) 2025-04-10
JP2025508959A5 JP2025508959A5 (https=) 2026-03-10

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ID=87884356

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JP2024552157A Pending JP2025508959A (ja) 2022-03-02 2023-03-01 Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法

Country Status (4)

Country Link
US (1) US20250197856A1 (https=)
EP (1) EP4486893A2 (https=)
JP (1) JP2025508959A (https=)
WO (1) WO2023168304A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1991677A2 (en) * 2006-01-26 2008-11-19 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
JP7714162B6 (ja) * 2018-12-03 2025-09-08 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド Ｍｓｈ３活性に関連するトリヌクレオチドリピート伸長障害の処置のための方法
KR20210106437A (ko) * 2018-12-20 2021-08-30 노파르티스 아게 3-(1-옥소이소인돌린-2-일)피페리딘-2,6-디온 유도체를 포함하는 투약 요법 및 약학적 조합물
AR122534A1 (es) * 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4247392A4 (en) * 2020-06-03 2025-10-08 Takeda Pharmaceuticals Usa Inc METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY

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Publication number Publication date
WO2023168304A3 (en) 2023-12-14
WO2023168304A2 (en) 2023-09-07
US20250197856A1 (en) 2025-06-19
EP4486893A2 (en) 2025-01-08

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