CL2019003337A1 - Oligonucleótidos antisentido para modular la expresión de htra1. - Google Patents

Oligonucleótidos antisentido para modular la expresión de htra1.

Info

Publication number
CL2019003337A1
CL2019003337A1 CL2019003337A CL2019003337A CL2019003337A1 CL 2019003337 A1 CL2019003337 A1 CL 2019003337A1 CL 2019003337 A CL2019003337 A CL 2019003337A CL 2019003337 A CL2019003337 A CL 2019003337A CL 2019003337 A1 CL2019003337 A1 CL 2019003337A1
Authority
CL
Chile
Prior art keywords
modulate
htra1
antisense oligonucleotides
expression
htra1 expression
Prior art date
Application number
CL2019003337A
Other languages
English (en)
Spanish (es)
Inventor
Søren Ottosen
Lykke Pedersen
Roberto Iacone
Peter Hagedorn
Susanne Kammler
Sindri Traustason
Rubén Alvarez Sánchez
Heidi Rye Hudlebusch
Marco Berrera
Andreas Dieckmann
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 CL2019003337A1 publication Critical patent/CL2019003337A1/es

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    • 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
    • C12N15/1137Non-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
    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21108HtrA2 peptidase (3.4.21.108)
    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CL2019003337A 2017-06-01 2019-11-20 Oligonucleótidos antisentido para modular la expresión de htra1. CL2019003337A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP17173964 2017-06-01
EP17209407 2017-12-21
EP17209535 2017-12-21

Publications (1)

Publication Number Publication Date
CL2019003337A1 true CL2019003337A1 (es) 2020-05-15

Family

ID=62245334

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019003337A CL2019003337A1 (es) 2017-06-01 2019-11-20 Oligonucleótidos antisentido para modular la expresión de htra1.

Country Status (19)

Country Link
US (3) US20190055564A1 (OSRAM)
EP (1) EP3630976A1 (OSRAM)
JP (1) JP7169995B2 (OSRAM)
KR (1) KR20200015608A (OSRAM)
CN (1) CN110691849A (OSRAM)
AU (1) AU2018277219A1 (OSRAM)
BR (1) BR112019025290A2 (OSRAM)
CA (1) CA3062590A1 (OSRAM)
CL (1) CL2019003337A1 (OSRAM)
CO (1) CO2019013317A2 (OSRAM)
CR (1) CR20190543A (OSRAM)
IL (1) IL271039A (OSRAM)
MA (1) MA49278A (OSRAM)
MX (1) MX2019014187A (OSRAM)
PE (1) PE20191845A1 (OSRAM)
PH (1) PH12019502636A1 (OSRAM)
RU (1) RU2019143004A (OSRAM)
TW (1) TW201907008A (OSRAM)
WO (1) WO2018220034A1 (OSRAM)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201803990A (zh) 2016-07-01 2018-02-01 赫孚孟拉羅股份公司 用於調節htra1表現之反股寡核苷酸
US11332733B2 (en) 2018-02-12 2022-05-17 lonis Pharmaceuticals, Inc. Modified compounds and uses thereof
JP2023539474A (ja) * 2020-08-20 2023-09-14 ジノム オピニオン インコーポレイテッド 加齢黄斑変性診断用バイオマーカーおよびこの用途
KR20230095097A (ko) * 2020-10-30 2023-06-28 히데아키 하라 눈 조직 섬유화의 저해에 사용하기 위한 gdf15 조절 물질

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JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
AU5290499A (en) * 1998-08-03 2000-02-28 Novartis Ag Human htra serine protease
ATE287897T2 (de) 1999-02-12 2005-02-15 Sankyo Co Analoga von nukleosiden und oligonukleotiden
CN102180924A (zh) 1999-05-04 2011-09-14 桑塔里斯制药公司 L-核糖-lna类似物
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
CA2506576C (en) 2002-11-18 2018-03-06 Santaris Pharma A/S Antisense gapmer oligonucleotides
CA2609180A1 (en) * 2005-05-24 2006-11-30 Isis Pharmaceuticals, Inc. Modulation of lmw-ptpase expression
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ, インコーポレーテッド 6−修飾された二環式核酸類似体
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
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US20110052602A1 (en) * 2006-07-26 2011-03-03 Yale University Diagnosis and Treatment of Age Related Macular Degeneration
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TW200911290A (en) * 2007-07-02 2009-03-16 Alcon Res Ltd RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration
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Also Published As

Publication number Publication date
WO2018220034A1 (en) 2018-12-06
TW201907008A (zh) 2019-02-16
US20190055564A1 (en) 2019-02-21
KR20200015608A (ko) 2020-02-12
PH12019502636A1 (en) 2020-06-08
CO2019013317A2 (es) 2020-01-17
EP3630976A1 (en) 2020-04-08
BR112019025290A2 (pt) 2020-06-23
RU2019143004A (ru) 2021-07-09
CR20190543A (es) 2020-02-10
JP7169995B2 (ja) 2022-11-11
JP2020521491A (ja) 2020-07-27
CA3062590A1 (en) 2018-12-06
PE20191845A1 (es) 2019-12-31
IL271039A (en) 2020-01-30
MA49278A (fr) 2020-04-08
CN110691849A (zh) 2020-01-14
MX2019014187A (es) 2020-01-27
US20200157546A1 (en) 2020-05-21
AU2018277219A1 (en) 2019-12-19
US20220042022A1 (en) 2022-02-10

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