AU2022293669A1 - Treatment of mtres1 related diseases and disorders - Google Patents

Treatment of mtres1 related diseases and disorders Download PDF

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Publication number
AU2022293669A1
AU2022293669A1 AU2022293669A AU2022293669A AU2022293669A1 AU 2022293669 A1 AU2022293669 A1 AU 2022293669A1 AU 2022293669 A AU2022293669 A AU 2022293669A AU 2022293669 A AU2022293669 A AU 2022293669A AU 2022293669 A1 AU2022293669 A1 AU 2022293669A1
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Australia
Prior art keywords
oligonucleotide
modified
measurement
composition
purines
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Pending
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AU2022293669A
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Inventor
Shannon BRUSE
Paul BUSKE
Brian CAJES
Omri GOTTESMAN
David JAKUBOSKY
Sarah KLEINSTEIN
David Lewis
David Rozema
John VEKICH
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Empirico Inc
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Empirico Inc
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Publication of AU2022293669A1 publication Critical patent/AU2022293669A1/en
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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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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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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    • C12N2320/00—Applications; Uses
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2022293669A 2021-06-16 2022-06-14 Treatment of mtres1 related diseases and disorders Pending AU2022293669A1 (en)

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
AU2022293669A1 true AU2022293669A1 (en) 2024-01-18

Family

ID=84527350

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022293669A Pending AU2022293669A1 (en) 2021-06-16 2022-06-14 Treatment of mtres1 related diseases and disorders

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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

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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
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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