JP7795914B2 - 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 - Google Patents

神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法

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JP7795914B2
JP7795914B2 JP2021571911A JP2021571911A JP7795914B2 JP 7795914 B2 JP7795914 B2 JP 7795914B2 JP 2021571911 A JP2021571911 A JP 2021571911A JP 2021571911 A JP2021571911 A JP 2021571911A JP 7795914 B2 JP7795914 B2 JP 7795914B2
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stmn2
oligonucleotide
linkage
qsn
seq
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JP2022536085A (ja
JP2022536085A5 (enExample
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ヒンクリー，サンドラ
ブラウン，ダンカン
アグラワル，スディール
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クラリス コーポレーション
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    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • 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 ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2021571911A 2019-06-03 2020-06-03 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 Active JP7795914B2 (ja)

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JP2025133172A JP2026002847A (ja) 2019-06-03 2025-08-08 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法

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US201962856264P 2019-06-03 2019-06-03
US62/856,264 2019-06-03
US201962914252P 2019-10-11 2019-10-11
US62/914,252 2019-10-11
US201962949817P 2019-12-18 2019-12-18
US62/949,817 2019-12-18
PCT/US2020/035811 WO2020247419A2 (en) 2019-06-03 2020-06-03 Oligonucleotides and methods of use for treating neurological diseases

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JP2025133172A Pending JP2026002847A (ja) 2019-06-03 2025-08-08 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法

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US (1) US20220333105A1 (enExample)
EP (1) EP3976010A4 (enExample)
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KR (1) KR20220033472A (enExample)
CN (1) CN114555069A (enExample)
AU (1) AU2020288555A1 (enExample)
BR (1) BR112021024463A2 (enExample)
CA (1) CA3142526A1 (enExample)
IL (1) IL288574A (enExample)
MX (1) MX2021014868A (enExample)
PH (1) PH12021553024A1 (enExample)
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US12496327B2 (en) 2019-01-14 2025-12-16 President And Fellows Of Harvard College Methods and compositions for restoring STMN2 levels
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WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
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WO2022216759A1 (en) * 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy
KR20240052763A (ko) * 2021-08-11 2024-04-23 아버 바이오테크놀로지스, 인크. 스타스민 2 (stmn2)를 표적화하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
CA3231484A1 (en) 2021-10-01 2023-04-06 Qinwen Mao Pathologic tdp-43 as a biomarker for the diagnosis of tdp-43 proteinopathy
MX2024006612A (es) * 2021-12-03 2024-06-12 Quralis Corp Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas.
GB202208384D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
EP4630558A2 (en) * 2022-12-09 2025-10-15 Auttx, LLC Targeted treatment of spliceopathy-induced neurological disorders
US20260232844A1 (en) * 2023-02-15 2026-08-13 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript
WO2025184347A1 (en) * 2024-02-27 2025-09-04 Emory University Network analysis of the cerebrospinal fluid proteome of sporadic and familial forms of amyotrophic lateral sclerosis and methods of treatment of the same
WO2026112528A1 (en) * 2024-11-22 2026-05-28 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof

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KR20220033472A (ko) 2022-03-16
WO2020247419A2 (en) 2020-12-10
EP3976010A4 (en) 2023-08-02
JP2026002847A (ja) 2026-01-08
IL288574A (en) 2022-02-01
JP2022536085A (ja) 2022-08-12
MX2021014868A (es) 2022-03-25
AU2020288555A1 (en) 2022-01-20
EP3976010A2 (en) 2022-04-06
BR112021024463A2 (pt) 2022-03-08
WO2020247419A3 (en) 2021-01-14
CN114555069A (zh) 2022-05-27
US20220333105A1 (en) 2022-10-20
CA3142526A1 (en) 2020-12-10
PH12021553024A1 (en) 2023-08-23

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