MA51634A - Oligonucléotides antisens d'alpha-synucléine et leurs utilisations - Google Patents

Oligonucléotides antisens d'alpha-synucléine et leurs utilisations

Info

Publication number
MA51634A
MA51634A MA051634A MA51634A MA51634A MA 51634 A MA51634 A MA 51634A MA 051634 A MA051634 A MA 051634A MA 51634 A MA51634 A MA 51634A MA 51634 A MA51634 A MA 51634A
Authority
MA
Morocco
Prior art keywords
synuclein
alpha
antisens
oligonucleotides
antisens oligonucleotides
Prior art date
Application number
MA051634A
Other languages
English (en)
Inventor
Jeffrey M Brown
Angela M Cacace
Martin Gill
Peter Hagedorn
Marianne Lerbech Jensen
Ivar M Macdonald
Jr Jere E Meredith
Richard E Olson
Annapurna Pendri
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 MA51634A publication Critical patent/MA51634A/fr

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Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide

Landscapes

  • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
MA051634A 2018-01-12 2019-01-11 Oligonucléotides antisens d'alpha-synucléine et leurs utilisations MA51634A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862616944P 2018-01-12 2018-01-12

Publications (1)

Publication Number Publication Date
MA51634A true MA51634A (fr) 2020-11-18

Family

ID=67219416

Family Applications (1)

Application Number Title Priority Date Filing Date
MA051634A MA51634A (fr) 2018-01-12 2019-01-11 Oligonucléotides antisens d'alpha-synucléine et leurs utilisations

Country Status (18)

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

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EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
MX2020013930A (es) 2018-06-22 2021-03-09 Hoffmann La Roche Oligonucleotidos para modular la expresion del canal de sodio dependiente de voltaje alfa subunidad 9 (scn9a).
BR112021000538A2 (pt) 2018-07-13 2021-04-06 F. Hoffmann-La Roche Ag Oligonucleotídeos para modular a expressão de rtel1
US20210214727A1 (en) * 2019-12-20 2021-07-15 Hoffmann-La Roche Inc. Enhanced oligonucleotides for inhibiting scn9a expression
AU2021206182A1 (en) * 2020-01-06 2022-07-28 AUM LifeTech, Inc. Antisense oligonucleotides for treatment of neurological disorders
CA3193830A1 (fr) * 2020-10-01 2022-04-07 Mangala Meenakshi Soundarapandian Compositions a base d'arni de snca et leurs procedes d'utilisation pour traiter ou prevenir des maladies neurodegeneratives associees a snca
PE20240696A1 (es) * 2021-03-08 2024-04-10 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina
CN118526514A (zh) * 2022-10-26 2024-08-23 武汉科技大学 一种促进缺血性脑卒中后血管新生的核酸药物及其应用
CN121287919B (zh) * 2025-12-03 2026-03-03 华南理工大学 α-突触核蛋白的表达调控及其应用

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Also Published As

Publication number Publication date
EP3737759A1 (fr) 2020-11-18
CN112424353A (zh) 2021-02-26
AU2019207859A1 (en) 2020-07-02
US20220119811A1 (en) 2022-04-21
IL275950A (en) 2020-08-31
JP2021511027A (ja) 2021-05-06
SG11202006142PA (en) 2020-07-29
RU2020126575A3 (fr) 2022-02-14
MX2020006973A (es) 2020-09-09
BR112020012921A2 (pt) 2020-12-08
CL2020001810A1 (es) 2020-11-27
JP2022130597A (ja) 2022-09-06
PH12020500570A1 (en) 2021-05-10
CR20200301A (es) 2020-10-26
AU2022224819A1 (en) 2022-09-29
KR20200109338A (ko) 2020-09-22
PE20210172A1 (es) 2021-01-29
CA3085964A1 (fr) 2019-07-18
CO2020008988A2 (es) 2020-08-31
WO2019138057A1 (fr) 2019-07-18
RU2020126575A (ru) 2022-02-14

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