MX2020006973A - Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos. - Google Patents

Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos.

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Publication number
MX2020006973A
MX2020006973A MX2020006973A MX2020006973A MX2020006973A MX 2020006973 A MX2020006973 A MX 2020006973A MX 2020006973 A MX2020006973 A MX 2020006973A MX 2020006973 A MX2020006973 A MX 2020006973A MX 2020006973 A MX2020006973 A MX 2020006973A
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MX
Mexico
Prior art keywords
antisense oligonucleotides
alpha
synuclein
snca
synuclein antisense
Prior art date
Application number
MX2020006973A
Other languages
English (en)
Inventor
Annapurna Pendri
Ivar M Mcdonald
Richard E Olson
Angela M Cacace
Peter Hagedorn
Marianne Lerbech Jensen
Jeffrey M Brown
Jr Jere E Meredith
Martin Gill
Original Assignee
Roche Innovation Ct Copenhagen As
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 Roche Innovation Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Publication of MX2020006973A publication Critical patent/MX2020006973A/es

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    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
    • C12N2310/323—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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    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

La presente descripción se refiere a oligonucleótidos antisentido, que se dirigen al transcrito de alfa-sinucleína (SNCA) en una célula, lo que da lugar a una expresión reducida de proteína SNCA. La reducción de la expresión de proteína SNCA es beneficiosa para el tratamiento de ciertos trastornos médicos, por ejemplo, un trastorno neurológico, tal como una sinucleinopatía.
MX2020006973A 2018-01-12 2019-01-11 Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos. MX2020006973A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616944P 2018-01-12 2018-01-12
PCT/EP2019/050661 WO2019138057A1 (en) 2018-01-12 2019-01-11 Alpha-synuclein antisense oligonucleotides and uses thereof

Publications (1)

Publication Number Publication Date
MX2020006973A true MX2020006973A (es) 2020-09-09

Family

ID=67219416

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020006973A MX2020006973A (es) 2018-01-12 2019-01-11 Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos.

Country Status (18)

Country Link
US (1) US20220119811A1 (es)
EP (1) EP3737759A1 (es)
JP (2) JP2021511027A (es)
KR (1) KR20200109338A (es)
CN (1) CN112424353A (es)
AU (2) AU2019207859A1 (es)
BR (1) BR112020012921A2 (es)
CA (1) CA3085964A1 (es)
CL (1) CL2020001810A1 (es)
CO (1) CO2020008988A2 (es)
CR (1) CR20200301A (es)
IL (1) IL275950A (es)
MA (1) MA51634A (es)
MX (1) MX2020006973A (es)
PE (1) PE20210172A1 (es)
PH (1) PH12020500570A1 (es)
SG (1) SG11202006142PA (es)
WO (1) WO2019138057A1 (es)

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US20220119811A1 (en) 2022-04-21
CA3085964A1 (en) 2019-07-18
CO2020008988A2 (es) 2020-08-31
JP2022130597A (ja) 2022-09-06
WO2019138057A1 (en) 2019-07-18
CR20200301A (es) 2020-10-26
AU2019207859A1 (en) 2020-07-02
SG11202006142PA (en) 2020-07-29
BR112020012921A2 (pt) 2020-12-08
PE20210172A1 (es) 2021-01-29
RU2020126575A3 (es) 2022-02-14
EP3737759A1 (en) 2020-11-18
AU2022224819A1 (en) 2022-09-29
KR20200109338A (ko) 2020-09-22
IL275950A (en) 2020-08-31
PH12020500570A1 (en) 2021-05-10
CL2020001810A1 (es) 2020-11-27
RU2020126575A (ru) 2022-02-14
CN112424353A (zh) 2021-02-26
MA51634A (fr) 2020-11-18
JP2021511027A (ja) 2021-05-06

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