JP2024529875A - 神経筋障害のためのオリゴヌクレオチドおよびその組成物 - Google Patents

神経筋障害のためのオリゴヌクレオチドおよびその組成物 Download PDF

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JP2024529875A
JP2024529875A JP2024501734A JP2024501734A JP2024529875A JP 2024529875 A JP2024529875 A JP 2024529875A JP 2024501734 A JP2024501734 A JP 2024501734A JP 2024501734 A JP2024501734 A JP 2024501734A JP 2024529875 A JP2024529875 A JP 2024529875A
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dux4
rna
administration
engineered
vector
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JP2024529875A5 (enExample
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アントニー・サーレハ
グラント・ベルガード
マートン・ムンズ
チャールズ・マルサク
ロバート・プレイス
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ミレキュール インコーポレイテッド
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/3125—Methylphosphonates
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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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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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

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2024501734A 2021-07-14 2022-07-14 神経筋障害のためのオリゴヌクレオチドおよびその組成物 Pending JP2024529875A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163221568P 2021-07-14 2021-07-14
US63/221,568 2021-07-14
PCT/US2022/073754 WO2023288289A2 (en) 2021-07-14 2022-07-14 Oligonucleotides and compositions thereof for neuromuscular disorders

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JP2024529875A true JP2024529875A (ja) 2024-08-14
JP2024529875A5 JP2024529875A5 (enExample) 2025-07-22

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US (1) US20240271134A1 (enExample)
EP (1) EP4370678A2 (enExample)
JP (1) JP2024529875A (enExample)
KR (1) KR20240032998A (enExample)
CN (1) CN117677698A (enExample)
AU (1) AU2022312501A1 (enExample)
CA (1) CA3223876A1 (enExample)
CO (1) CO2024000116A2 (enExample)
IL (1) IL310063A (enExample)
MX (1) MX2024000754A (enExample)
WO (1) WO2023288289A2 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
US20260159835A1 (en) * 2024-12-05 2026-06-11 Soufflé Therapeutics, Inc. Oligonucleotide-mediated knockdown of dux4

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013016352A1 (en) * 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2017007886A2 (en) * 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
CN116096890A (zh) 2020-04-02 2023-05-09 米雷楚莱股份有限公司 使用工程化寡核苷酸的靶向抑制
US20240336915A1 (en) * 2021-01-22 2024-10-10 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dux4 expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013016352A1 (en) * 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2017007886A2 (en) * 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LU-NGUYEN N. ET AL., HUM MOL GENET, vol. 30, JPN6026021960, 9 July 2021 (2021-07-09), pages 1398 - 1412, ISSN: 0005875585 *

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CO2024000116A2 (es) 2024-02-05
WO2023288289A2 (en) 2023-01-19
US20240271134A1 (en) 2024-08-15
KR20240032998A (ko) 2024-03-12
AU2022312501A1 (en) 2024-02-29
CN117677698A (zh) 2024-03-08
WO2023288289A3 (en) 2023-04-13
IL310063A (en) 2024-03-01
CA3223876A1 (en) 2023-01-19
EP4370678A2 (en) 2024-05-22
MX2024000754A (es) 2024-04-18

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