JP2024523265A - Ｍｔｒｅｓ１関連の疾患および障害の処置 - Google Patents

Ｍｔｒｅｓ１関連の疾患および障害の処置 Download PDF

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JP2024523265A
JP2024523265A JP2023576324A JP2023576324A JP2024523265A JP 2024523265 A JP2024523265 A JP 2024523265A JP 2023576324 A JP2023576324 A JP 2023576324A JP 2023576324 A JP2023576324 A JP 2023576324A JP 2024523265 A JP2024523265 A JP 2024523265A
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oligonucleotide
modified
composition
purines
pyrimidines
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JP2024523265A5 (https=
JPWO2022266045A5 (https=
Inventor
ゴッテスマン，オムリ
ブルース，シャノン
バスケ，ポール
カジェス，ブライアン
ジャクボウスキ，デイビッド
クラインシュタイン，サラ
ルイス，デイビッド
ロゼマ，デイビッド
ヴェキック，ジョン
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エンピリコ インク．
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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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023576324A 2021-06-16 2022-06-14 Ｍｔｒｅｓ１関連の疾患および障害の処置 Pending JP2024523265A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163211379P 2021-06-16 2021-06-16
US63/211,379 2021-06-16
PCT/US2022/033356 WO2022266045A1 (en) 2021-06-16 2022-06-14 Treatment of mtres1 related diseases and disorders

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JP2024523265A true JP2024523265A (ja) 2024-06-28
JP2024523265A5 JP2024523265A5 (https=) 2025-06-20
JPWO2022266045A5 JPWO2022266045A5 (https=) 2025-06-20

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US (1) US20240287518A1 (https=)
EP (1) EP4355878A4 (https=)
JP (1) JP2024523265A (https=)
KR (1) KR20240034185A (https=)
CN (1) CN117980478A (https=)
AU (1) AU2022293669A1 (https=)
CA (1) CA3221625A1 (https=)
IL (1) IL309317A (https=)
MX (1) MX2023015242A (https=)
WO (1) WO2022266045A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024091874A1 (en) * 2022-10-25 2024-05-02 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders
WO2024091873A2 (en) * 2022-10-25 2024-05-02 Empirico Inc. Small molecule treatment of mtres1 related diseases and disorders
WO2025158407A2 (en) * 2024-01-26 2025-07-31 Empirico Inc. Treatment of mtres1 related diseases and disorders
WO2025226835A1 (en) * 2024-04-24 2025-10-30 Empirico Inc. Gene therapy treatment of mtres1 related diseases and disorders
WO2025255269A1 (en) * 2024-06-05 2025-12-11 Empirico Inc. Treatment of mtres1 related diseases and disorders

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080279846A1 (en) * 2007-05-11 2008-11-13 Thomas Jefferson University Methods of treatment and prevention of neurodegenerative diseases and disorders
US10004814B2 (en) * 2013-11-11 2018-06-26 Sirna Therapeutics, Inc. Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
US10287580B2 (en) * 2014-03-14 2019-05-14 The General Hospital Corporation Molecular re-engineering of excitation-inhibition balance in memory circuits
PH12018501207B1 (en) * 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
EP4004206A1 (en) * 2019-07-23 2022-06-01 University of Rochester Targeted rna cleavage with crispr-cas

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EP4355878A4 (en) 2025-04-09
US20240287518A1 (en) 2024-08-29
KR20240034185A (ko) 2024-03-13
WO2022266045A1 (en) 2022-12-22
AU2022293669A1 (en) 2024-01-18
EP4355878A1 (en) 2024-04-24
MX2023015242A (es) 2024-03-11
IL309317A (en) 2024-02-01
CN117980478A (zh) 2024-05-03
CA3221625A1 (en) 2022-12-22

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