JP2023519871A - Ｓｔｍｎ２レベルを回復させるための方法及び組成物 - Google Patents

Ｓｔｍｎ２レベルを回復させるための方法及び組成物 Download PDF

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JP2023519871A
JP2023519871A JP2022558277A JP2022558277A JP2023519871A JP 2023519871 A JP2023519871 A JP 2023519871A JP 2022558277 A JP2022558277 A JP 2022558277A JP 2022558277 A JP2022558277 A JP 2022558277A JP 2023519871 A JP2023519871 A JP 2023519871A
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stmn2
tdp
seq
disease
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JP2023519871A5 (https=
JPWO2021195446A5 (https=
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ケビン シー． エガン，
ジョセフ ロバート クリム，
ロバート エイチ． ジュニア ブラウン，
ジョナサン ケー． ワッツ，
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Harvard University
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    • 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
    • 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
    • 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
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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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/316—Phosphonothioates
    • 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
    • C12N2310/32—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
    • 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
    • C12N2320/33—Alteration of splicing

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  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
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  • Biophysics (AREA)
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  • Hospice & Palliative Care (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (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)
JP2022558277A 2020-03-25 2021-03-25 Ｓｔｍｎ２レベルを回復させるための方法及び組成物 Pending JP2023519871A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025181104A JP2026027295A (ja) 2020-03-25 2025-10-27 Ｓｔｍｎ２レベルを回復させるための方法及び組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062994797P 2020-03-25 2020-03-25
US62/994,797 2020-03-25
US202063063174P 2020-08-07 2020-08-07
US63/063,174 2020-08-07
US202163133749P 2021-01-04 2021-01-04
US63/133,749 2021-01-04
PCT/US2021/024254 WO2021195446A2 (en) 2020-03-25 2021-03-25 Methods and compositions for restoring stmn2 levels

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JP2025181104A Division JP2026027295A (ja) 2020-03-25 2025-10-27 Ｓｔｍｎ２レベルを回復させるための方法及び組成物

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JP2023519871A true JP2023519871A (ja) 2023-05-15
JP2023519871A5 JP2023519871A5 (https=) 2024-04-03
JPWO2021195446A5 JPWO2021195446A5 (https=) 2024-04-03

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JP2025181104A Pending JP2026027295A (ja) 2020-03-25 2025-10-27 Ｓｔｍｎ２レベルを回復させるための方法及び組成物

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US (1) US20240011027A1 (https=)
EP (1) EP4127172A4 (https=)
JP (2) JP2023519871A (https=)
CN (1) CN116034161A (https=)
CA (1) CA3176884A1 (https=)
WO (1) WO2021195446A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023534557A (ja) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020150290A2 (en) 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels
JP7795914B2 (ja) * 2019-06-03 2026-01-08 クラリス コーポレーション 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
EP4162051A4 (en) * 2020-06-03 2025-07-09 Quralis Corp TREATMENT OF NEUROLOGICAL DISEASES USING GENE TRANSCRIPT MODULATORS
GB2617565A (en) * 2022-04-11 2023-10-18 Ucl Business Ltd A construct, vector and system and uses thereof
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
WO2024249458A2 (en) * 2023-05-31 2024-12-05 The Trustees Of Columbia University In The City Of New York Fine-tuning inflammation by altering 3'utr length
WO2025076126A1 (en) * 2023-10-02 2025-04-10 Emory University Agents that bind nucleic acids encoding elavl3 cryptic exons, pharmaceutical compositions, and uses in managing neurological diseases
CN118737269B (zh) * 2024-08-30 2024-11-19 墨卓生物科技(浙江)有限公司 在单细胞微生物基因组测序结果中区分菌株的方法

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019241648A1 (en) * 2018-06-14 2019-12-19 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing stmn2 expression
WO2020247419A2 (en) * 2019-06-03 2020-12-10 Quralis Corporation Oligonucleotides and methods of use for treating neurological diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019241648A1 (en) * 2018-06-14 2019-12-19 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing stmn2 expression
WO2020247419A2 (en) * 2019-06-03 2020-12-10 Quralis Corporation Oligonucleotides and methods of use for treating neurological diseases

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023534557A (ja) * 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド

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EP4127172A4 (en) 2025-06-04
JP2026027295A (ja) 2026-02-18
WO2021195446A3 (en) 2021-11-11
WO2021195446A2 (en) 2021-09-30
CA3176884A1 (en) 2021-09-30
EP4127172A2 (en) 2023-02-08
CN116034161A (zh) 2023-04-28
US20240011027A1 (en) 2024-01-11

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