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 (OSRAM
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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
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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)
  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (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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JP2022536085A5 JP2022536085A5 (OSRAM) 2023-06-06
JP7795914B2 true JP7795914B2 (ja) 2026-01-08

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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 (OSRAM)
EP (1) EP3976010A4 (OSRAM)
JP (2) JP7795914B2 (OSRAM)
KR (1) KR20220033472A (OSRAM)
CN (1) CN114555069A (OSRAM)
AU (1) AU2020288555A1 (OSRAM)
BR (1) BR112021024463A2 (OSRAM)
CA (1) CA3142526A1 (OSRAM)
IL (1) IL288574A (OSRAM)
MX (1) MX2021014868A (OSRAM)
PH (1) PH12021553024A1 (OSRAM)
WO (1) WO2020247419A2 (OSRAM)

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EP4127172A4 (en) * 2020-03-25 2025-06-04 President and Fellows of Harvard College Methods and compositions for restoring STMN2 levels
KR20230043819A (ko) * 2020-06-03 2023-03-31 큐랄리스 코포레이션 유전자 전사체의 조정제를 사용한 신경계 질환의 치료
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
AU2022255175A1 (en) * 2021-04-06 2023-11-23 Trace Neuroscience, Inc. Compositions and methods for treating tdp-43 proteinopathy
IL310616A (en) * 2021-08-11 2024-04-01 Arbor Biotechnologies Inc Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
CA3231484A1 (en) 2021-10-01 2023-04-06 Qinwen Mao Pathologic tdp-43 as a biomarker for the diagnosis of tdp-43 proteinopathy
AU2022402929A1 (en) * 2021-12-03 2024-06-13 Quralis Corporation Splice switcher antisense oligonucleotides with modified backbone chemistries
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
GB202208384D0 (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
CN120712355A (zh) * 2023-02-15 2025-09-26 阿伯生物技术公司 用于抑制抑微管装配蛋白2（stmn2）转录物中的异常剪接的基因编辑方法
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

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JP2016513976A (ja) 2013-03-14 2016-05-19 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タウ発現を調節するための組成物および方法
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MX2021014868A (es) 2022-03-25
JP2022536085A (ja) 2022-08-12
WO2020247419A3 (en) 2021-01-14
AU2020288555A1 (en) 2022-01-20
KR20220033472A (ko) 2022-03-16
EP3976010A2 (en) 2022-04-06
JP2026002847A (ja) 2026-01-08
PH12021553024A1 (en) 2023-08-23
CA3142526A1 (en) 2020-12-10
EP3976010A4 (en) 2023-08-02
BR112021024463A2 (pt) 2022-03-08
WO2020247419A2 (en) 2020-12-10
IL288574A (en) 2022-02-01
US20220333105A1 (en) 2022-10-20
CN114555069A (zh) 2022-05-27

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