MX2023001222A - Compuestos y metodos para reducir la expresion de app. - Google Patents

Compuestos y metodos para reducir la expresion de app.

Info

Publication number
MX2023001222A
MX2023001222A MX2023001222A MX2023001222A MX2023001222A MX 2023001222 A MX2023001222 A MX 2023001222A MX 2023001222 A MX2023001222 A MX 2023001222A MX 2023001222 A MX2023001222 A MX 2023001222A MX 2023001222 A MX2023001222 A MX 2023001222A
Authority
MX
Mexico
Prior art keywords
compounds
methods
reducing
animal
cell
Prior art date
Application number
MX2023001222A
Other languages
English (en)
Inventor
Thazha P Prakash
Holly Kordasiewicz
Huynh- Hoa Bui
Susan M Freier
W Brad Wan
Hien Thuy Zhao
Ruben E Valas
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of MX2023001222A publication Critical patent/MX2023001222A/es

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    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 N.A.
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/30—Chemical structure
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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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
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Public Health (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Medicinal Preparation (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Hospice & Palliative Care (AREA)
  • Saccharide Compounds (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)

Abstract

Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o actividad de ARN de APP en una célula o animal y, en determinados casos, reducir la cantidad de proteína APP en una célula o animal. Dichos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o signo de una enfermedad o trastorno neurodegenerativo. Dichos síntomas y signos incluyen deterioro cognitivo, que incluye declive de la memoria y de las habilidades del lenguaje, síntomas conductuales y psicológicos tales como apatía y falta de motivación, alteraciones en la marcha y convulsiones, demencia progresiva y depósitos amiloides anormales.
MX2023001222A 2020-07-28 2021-07-28 Compuestos y metodos para reducir la expresion de app. MX2023001222A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063057816P 2020-07-28 2020-07-28
US202063106616P 2020-10-28 2020-10-28
US202063129255P 2020-12-22 2020-12-22
US202163148514P 2021-02-11 2021-02-11
PCT/US2021/043520 WO2022026589A1 (en) 2020-07-28 2021-07-28 Compounds and methods for reducing app expression

Publications (1)

Publication Number Publication Date
MX2023001222A true MX2023001222A (es) 2023-04-26

Family

ID=80036715

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023001222A MX2023001222A (es) 2020-07-28 2021-07-28 Compuestos y metodos para reducir la expresion de app.

Country Status (12)

Country Link
US (1) US20230055405A1 (es)
EP (1) EP4189090A1 (es)
JP (1) JP2023536472A (es)
KR (1) KR20230043914A (es)
AU (1) AU2021315992A1 (es)
BR (1) BR112023001624A2 (es)
CA (1) CA3189922A1 (es)
IL (1) IL299686A (es)
MX (1) MX2023001222A (es)
TW (1) TW202221014A (es)
UY (1) UY39344A (es)
WO (1) WO2022026589A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
AU2023236560A1 (en) * 2022-03-17 2024-10-03 Aligos Therapeutics, Inc. Modified gapmer oligomers and methods of use thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3759127A4 (en) * 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
EP3898979A4 (en) * 2018-12-19 2022-10-12 Alnylam Pharmaceuticals, Inc. AMYLOID PROTEIN PRECURSOR (APP) RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF

Also Published As

Publication number Publication date
US20230055405A1 (en) 2023-02-23
UY39344A (es) 2022-02-25
WO2022026589A1 (en) 2022-02-03
AU2021315992A1 (en) 2023-02-09
TW202221014A (zh) 2022-06-01
EP4189090A1 (en) 2023-06-07
BR112023001624A2 (pt) 2023-02-23
KR20230043914A (ko) 2023-03-31
CA3189922A1 (en) 2022-02-03
IL299686A (en) 2023-03-01
JP2023536472A (ja) 2023-08-25

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