KR20240034185A - Mtres1 관련 질환 및 장애의 치료 - Google Patents

Mtres1 관련 질환 및 장애의 치료 Download PDF

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Publication number
KR20240034185A
KR20240034185A KR1020247000882A KR20247000882A KR20240034185A KR 20240034185 A KR20240034185 A KR 20240034185A KR 1020247000882 A KR1020247000882 A KR 1020247000882A KR 20247000882 A KR20247000882 A KR 20247000882A KR 20240034185 A KR20240034185 A KR 20240034185A
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South Korea
Prior art keywords
modified
oligonucleotide
composition
pyrimidines
purines
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Pending
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KR1020247000882A
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English (en)
Korean (ko)
Inventor
옴리 갓스만
섀논 브루스
폴 버스크
브라이언 카제스
데이비드 자쿠보스키
사라 클라인스타인
데이비드 루이스
데이비드 로제마
존 베키치
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엠피리코 인크.
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Publication of KR20240034185A publication Critical patent/KR20240034185A/ko
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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
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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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    • C12N2310/32—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
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
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  • Neurology (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247000882A 2021-06-16 2022-06-14 Mtres1 관련 질환 및 장애의 치료 Pending KR20240034185A (ko)

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
KR20240034185A true KR20240034185A (ko) 2024-03-13

Family

ID=84527350

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247000882A Pending KR20240034185A (ko) 2021-06-16 2022-06-14 Mtres1 관련 질환 및 장애의 치료

Country Status (10)

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
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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Patent event date: 20240109

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Patent event code: PA02012R01D

Patent event date: 20250613

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