CN117980478A - Mtres1相关疾病和病症的治疗 - Google Patents

Mtres1相关疾病和病症的治疗 Download PDF

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Publication number
CN117980478A
CN117980478A CN202280056109.XA CN202280056109A CN117980478A CN 117980478 A CN117980478 A CN 117980478A CN 202280056109 A CN202280056109 A CN 202280056109A CN 117980478 A CN117980478 A CN 117980478A
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China
Prior art keywords
modified
oligonucleotide
composition
purines
sense strand
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Pending
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CN202280056109.XA
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English (en)
Chinese (zh)
Inventor
奥马里·戈特斯曼
香农·布鲁斯
保罗·布斯克
布赖恩·加耶斯
大卫·雅库伯斯基
莎拉·克莱因施泰因
大卫·路易斯
大卫·罗泽马
约翰·维基奇
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Eperik Co
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Eperik Co
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Publication of CN117980478A publication Critical patent/CN117980478A/zh
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • 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
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    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12N2310/30—Chemical structure
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    • C12N2310/3521—Methyl
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  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202280056109.XA 2021-06-16 2022-06-14 Mtres1相关疾病和病症的治疗 Pending CN117980478A (zh)

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

Publications (1)

Publication Number Publication Date
CN117980478A true CN117980478A (zh) 2024-05-03

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ID=84527350

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280056109.XA Pending CN117980478A (zh) 2021-06-16 2022-06-14 Mtres1相关疾病和病症的治疗

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Country Link
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

Also Published As

Publication number Publication date
JP2024523265A (ja) 2024-06-28
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
CA3221625A1 (en) 2022-12-22

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