CO2018013240A2 - Oligonucleótidos antisentido para modular la expresión de htra1 - Google Patents

Oligonucleótidos antisentido para modular la expresión de htra1

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Publication number
CO2018013240A2
CO2018013240A2 CONC2018/0013240A CO2018013240A CO2018013240A2 CO 2018013240 A2 CO2018013240 A2 CO 2018013240A2 CO 2018013240 A CO2018013240 A CO 2018013240A CO 2018013240 A2 CO2018013240 A2 CO 2018013240A2
Authority
CO
Colombia
Prior art keywords
antisense oligonucleotides
modulate
htra1
htra1 expression
expression
Prior art date
Application number
CONC2018/0013240A
Other languages
English (en)
Inventor
Roberto Iacone
Peter Hagedorn
Susanne Kammler
Søren Ottosen
Sindri Traustason
Heidi Hudlebusch
Lykke Pedersen
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 CO2018013240A2 publication Critical patent/CO2018013240A2/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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/30—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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/34—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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invención se refiere a oligonucleótidos (oligómeros) antisentido que son complementarios a HTRA1, que conducen a la modulación de la expresión de HTRA1. La modulación de la expresión de HTRA1 resulta beneficiosa para un abanico de trastornos médicos, tales como la degeneración macular, por ejemplo la degeneración macular asociada a la edad.
CONC2018/0013240A 2016-07-01 2018-12-06 Oligonucleótidos antisentido para modular la expresión de htra1 CO2018013240A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16177508 2016-07-01
EP17170129 2017-05-09
PCT/EP2017/065937 WO2018002105A1 (en) 2016-07-01 2017-06-28 Antisense oligonucleotides for modulating htra1 expression

Publications (1)

Publication Number Publication Date
CO2018013240A2 true CO2018013240A2 (es) 2018-12-14

Family

ID=59295175

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2018/0013240A CO2018013240A2 (es) 2016-07-01 2018-12-06 Oligonucleótidos antisentido para modular la expresión de htra1

Country Status (21)

Country Link
US (2) US10519450B2 (es)
EP (1) EP3478839A1 (es)
JP (3) JP7060525B2 (es)
KR (1) KR20190025970A (es)
CN (1) CN109415732B (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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EP4257191A3 (en) 2016-06-21 2023-11-22 Orion Ophthalmology LLC Heterocyclic prolinamide derivatives
JP7164521B2 (ja) 2016-06-21 2022-11-01 オリオン・オフサルモロジー・エルエルシー 炭素環式プロリンアミド誘導体
US10519450B2 (en) 2016-07-01 2019-12-31 Hoffmann-La Roche Inc. Antisense oligonucleotides for modulating HTRA1 expression
US11597926B2 (en) 2017-12-22 2023-03-07 Roche Innovation Center Copenhagen A/S Thiophosphoramidites
KR20200104345A (ko) 2017-12-22 2020-09-03 로슈 이노베이션 센터 코펜하겐 에이/에스 포스포로디티오에이트 뉴클레오시드간 연결을 포함하는 올리고뉴클레오티드
AU2018386527A1 (en) 2017-12-22 2020-04-30 Roche Innovation Center Copenhagen A/S Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2020007772A1 (en) * 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020109344A1 (en) * 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Occular administration device for antisense oligonucleotides
WO2020109343A1 (en) * 2018-11-29 2020-06-04 F. Hoffmann-La Roche Ag Combination therapy for treatment of macular degeneration

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Also Published As

Publication number Publication date
SG10202005885VA (en) 2020-07-29
CR20180606A (es) 2019-02-14
PH12018502707A1 (en) 2019-11-11
JP2022106785A (ja) 2022-07-20
CN109415732A (zh) 2019-03-01
TW201803990A (zh) 2018-02-01
KR20190025970A (ko) 2019-03-12
CL2018003698A1 (es) 2019-04-05
PE20190381A1 (es) 2019-03-08
MA45620A (fr) 2019-05-08
US10519450B2 (en) 2019-12-31
BR112018077321A2 (pt) 2019-04-24
CA3026855A1 (en) 2018-01-04
RU2019101298A (ru) 2020-08-03
WO2018002105A1 (en) 2018-01-04
US20180002701A1 (en) 2018-01-04
EP3478839A1 (en) 2019-05-08
AU2017289204A1 (en) 2019-01-17
US20200399641A1 (en) 2020-12-24
JP2019522987A (ja) 2019-08-22
MX2018015722A (es) 2019-05-27
JP7416852B2 (ja) 2024-01-17
SG11201811554SA (en) 2019-01-30
IL263489A (en) 2019-01-31
JP7060525B2 (ja) 2022-04-26
CN109415732B (zh) 2024-01-02
ZA201808291B (en) 2019-10-30
JP2024029178A (ja) 2024-03-05
RU2019101298A3 (es) 2021-01-26

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