CO2020008988A2 - Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos - Google Patents

Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos

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Publication number
CO2020008988A2
CO2020008988A2 CONC2020/0008988A CO2020008988A CO2020008988A2 CO 2020008988 A2 CO2020008988 A2 CO 2020008988A2 CO 2020008988 A CO2020008988 A CO 2020008988A CO 2020008988 A2 CO2020008988 A2 CO 2020008988A2
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CO
Colombia
Prior art keywords
synuclein
alpha
antisense oligonucleotides
snca
expression
Prior art date
Application number
CONC2020/0008988A
Other languages
English (en)
Inventor
Peter Hagedorn
Jeffrey M Brown
Martin Gill
Angela M Cacace
Marianne Lerbech Jensen
Ivar M Mcdonald
Richard E Olson
Annapurna Pendri
Jr Jere E Meredith
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 CO2020008988A2 publication Critical patent/CO2020008988A2/es

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/30—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)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (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 divulgación 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.
CONC2020/0008988A 2018-01-12 2020-07-22 Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos CO2020008988A2 (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
CO2020008988A2 true CO2020008988A2 (es) 2020-08-31

Family

ID=67219416

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0008988A CO2020008988A2 (es) 2018-01-12 2020-07-22 Oligonucleótidos antisentido para alfa-sinucleína 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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MX2023010535A (es) * 2021-03-08 2023-09-19 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina.

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BR112020012921A2 (pt) 2020-12-08
RU2020126575A (ru) 2022-02-14
JP2021511027A (ja) 2021-05-06
CA3085964A1 (en) 2019-07-18
JP2022130597A (ja) 2022-09-06
EP3737759A1 (en) 2020-11-18
WO2019138057A1 (en) 2019-07-18
AU2022224819A1 (en) 2022-09-29
PH12020500570A1 (en) 2021-05-10
CL2020001810A1 (es) 2020-11-27
RU2020126575A3 (es) 2022-02-14
AU2019207859A1 (en) 2020-07-02
CR20200301A (es) 2020-10-26
MX2020006973A (es) 2020-09-09
US20220119811A1 (en) 2022-04-21
CN112424353A (zh) 2021-02-26
SG11202006142PA (en) 2020-07-29
PE20210172A1 (es) 2021-01-29
MA51634A (fr) 2020-11-18
IL275950A (en) 2020-08-31
KR20200109338A (ko) 2020-09-22

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