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PE20190381A1 - Oligonucleotidos antisentido para modular la expresion de htra1 - Google Patents

Oligonucleotidos antisentido para modular la expresion de htra1

Info

Publication number
PE20190381A1
PE20190381A1 PE2018003308A PE2018003308A PE20190381A1 PE 20190381 A1 PE20190381 A1 PE 20190381A1 PE 2018003308 A PE2018003308 A PE 2018003308A PE 2018003308 A PE2018003308 A PE 2018003308A PE 20190381 A1 PE20190381 A1 PE 20190381A1
Authority
PE
Peru
Prior art keywords
htra1
expression
modulate
sense oligonucleotides
refers
Prior art date
Application number
PE2018003308A
Other languages
English (en)
Inventor
Peter Hagedorn
Susanne Kammler
Søren Ottosen
Sindri Traustason
Heidi Rye Hudlebusch
Lykke Pedersen
Roberto Iacone
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of PE20190381A1 publication Critical patent/PE20190381A1/es

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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
    • C12N15/1137—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 against enzymes
    • 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
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • 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
    • C12N2310/30—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
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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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)

Abstract

SE REFIERE A UN OLIGONUCLEOTIDO ANTISENTIDO DE FORMULA (I) DE SEQ ID NO: 143 Y A UNA SAL FARMACEUTICAMENTE ACEPTABLE DEL MISMO TAL COMO LA SAL SODICA O LA SAL POTASICA. TAMBIEN SE REFIERE A UNA COMPOSICION FARMACEUTICA QUE COMPRENDE DICHO OLIGONUCLEOTIDO ANTISENTIDO, EL CUAL MODULA LA EXPRESION DE HTRA1 SIENDO UTIL PARA TRATAR O PREVENIR LA DEGENERACION MACULAR ASOCIADA A LA EDAD
PE2018003308A 2016-07-01 2017-06-28 Oligonucleotidos antisentido para modular la expresion de htra1 PE20190381A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP16177508 2016-07-01
EP17170129 2017-05-09

Publications (1)

Publication Number Publication Date
PE20190381A1 true PE20190381A1 (es) 2019-03-08

Family

ID=59295175

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2018003308A PE20190381A1 (es) 2016-07-01 2017-06-28 Oligonucleotidos antisentido para modular la expresion de htra1

Country Status (21)

Country Link
US (2) US10519450B2 (es)
EP (1) EP3478839A1 (es)
JP (3) JP7060525B2 (es)
KR (1) KR20190025970A (es)
CN (2) CN118325897A (es)
AU (1) AU2017289204A1 (es)
BR (1) BR112018077321A2 (es)
CA (1) CA3026855A1 (es)
CL (1) CL2018003698A1 (es)
CO (1) CO2018013240A2 (es)
CR (1) CR20180606A (es)
IL (1) IL263489A (es)
MA (1) MA45620A (es)
MX (1) MX2018015722A (es)
PE (1) PE20190381A1 (es)
PH (1) PH12018502707A1 (es)
RU (1) RU2019101298A (es)
SG (2) SG10202005885VA (es)
TW (1) TW201803990A (es)
WO (1) WO2018002105A1 (es)
ZA (1) ZA201808291B (es)

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Also Published As

Publication number Publication date
SG11201811554SA (en) 2019-01-30
MA45620A (fr) 2019-05-08
JP7060525B2 (ja) 2022-04-26
CN118325897A (zh) 2024-07-12
US10519450B2 (en) 2019-12-31
CA3026855A1 (en) 2018-01-04
EP3478839A1 (en) 2019-05-08
CR20180606A (es) 2019-02-14
ZA201808291B (en) 2019-10-30
RU2019101298A (ru) 2020-08-03
JP2019522987A (ja) 2019-08-22
JP7416852B2 (ja) 2024-01-17
JP2024029178A (ja) 2024-03-05
KR20190025970A (ko) 2019-03-12
US20200399641A1 (en) 2020-12-24
RU2019101298A3 (es) 2021-01-26
IL263489A (en) 2019-01-31
PH12018502707A1 (en) 2019-11-11
SG10202005885VA (en) 2020-07-29
TW201803990A (zh) 2018-02-01
WO2018002105A1 (en) 2018-01-04
JP2022106785A (ja) 2022-07-20
BR112018077321A2 (pt) 2019-04-24
CN109415732A (zh) 2019-03-01
AU2017289204A1 (en) 2019-01-17
MX2018015722A (es) 2019-05-27
CO2018013240A2 (es) 2018-12-14
CL2018003698A1 (es) 2019-04-05
CN109415732B (zh) 2024-01-02
US20180002701A1 (en) 2018-01-04

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