BR112019025290A2 - Oligonucleotídeos anti-sentido para modulação da expressão de htra1 - Google Patents

Oligonucleotídeos anti-sentido para modulação da expressão de htra1 Download PDF

Info

Publication number
BR112019025290A2
BR112019025290A2 BR112019025290-8A BR112019025290A BR112019025290A2 BR 112019025290 A2 BR112019025290 A2 BR 112019025290A2 BR 112019025290 A BR112019025290 A BR 112019025290A BR 112019025290 A2 BR112019025290 A2 BR 112019025290A2
Authority
BR
Brazil
Prior art keywords
oligonucleotide
nucleosides
seq
htra1
region
Prior art date
Application number
BR112019025290-8A
Other languages
English (en)
Portuguese (pt)
Inventor
Alvarez Sánchez Rubén
Rubén Alvarez Sánchez
Iacone Roberto
Andreas Dieckmann
Hagedorn Peter
Roberto Iacone
Kammler Susanne
Peter Hagedorn
Ottosen Søren
Susanne Kammler
Traustason Sindri
Søren OTTOSEN
Rye Hudlebusch Heidi
Sindri Traustason
PEDERSEN Lykke
Heidi Rye Hudlebusch
Berrera Marco
Lykke Pedersen
Dieckmann Andreas
Marco Berrera
Original Assignee
F. Hoffmann-La Roche Ag
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 F. Hoffmann-La Roche Ag filed Critical F. Hoffmann-La Roche Ag
Publication of BR112019025290A2 publication Critical patent/BR112019025290A2/pt

Links

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
    • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
BR112019025290-8A 2017-06-01 2018-05-30 Oligonucleotídeos anti-sentido para modulação da expressão de htra1 BR112019025290A2 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
EP17173964.2 2017-06-01
EP17173964 2017-06-01
EP17209407.0 2017-12-21
EP17209535.8 2017-12-21
EP17209535 2017-12-21
EP17209407 2017-12-21
PCT/EP2018/064221 WO2018220034A1 (en) 2017-06-01 2018-05-30 Antisense oligonucleotides for modulating htra1 expression

Publications (1)

Publication Number Publication Date
BR112019025290A2 true BR112019025290A2 (pt) 2020-06-23

Family

ID=62245334

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019025290-8A BR112019025290A2 (pt) 2017-06-01 2018-05-30 Oligonucleotídeos anti-sentido para modulação da expressão de htra1

Country Status (19)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017289204A1 (en) 2016-07-01 2019-01-17 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating HTRA1 expression
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
CN116507741A (zh) * 2020-08-20 2023-07-28 基因组评论股份有限公司 年龄相关性黄斑变性诊断用生物标记物及其用途
CA3199951A1 (en) * 2020-10-30 2022-05-05 Hideaki Hara Gdf15 modulators for use in inhibiting ocular tissue fibrosis

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
WO2000008134A2 (en) * 1998-08-03 2000-02-17 Novartis Ag HUMAN HtrA SERINE PROTEASE
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US9045518B2 (en) 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
EP2462937A1 (en) * 2005-05-24 2012-06-13 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
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007277125B2 (en) * 2006-07-26 2013-08-01 Yale University Diagnosis and treatment of age related macular degeneration
EP2081596A4 (en) * 2006-10-06 2010-07-21 Univ Utah Res Found METHOD FOR THE DETECTION OF EYE DISEASES AND PATHOLOGICAL CONDITIONS AND TREATMENT THEREFOR
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
TW200911290A (en) 2007-07-02 2009-03-16 Alcon Res Ltd RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2217720A4 (en) * 2007-11-01 2010-12-08 Univ Iowa Res Found RNA LOCUS ANALYSIS TO ASSESS THE STUNNING FOR AMD AND MPGNII
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
FR2965278B1 (fr) * 2010-09-23 2014-10-10 Univ Caen Basse Normandie Procede d'obtention in vitro ou ex vivo de chondrocytes et leurs utilisations
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
US20190284621A1 (en) * 2016-11-11 2019-09-19 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection

Also Published As

Publication number Publication date
KR20200015608A (ko) 2020-02-12
TW201907008A (zh) 2019-02-16
JP7169995B2 (ja) 2022-11-11
CN110691849A (zh) 2020-01-14
EP3630976A1 (en) 2020-04-08
CR20190543A (es) 2020-02-10
PH12019502636A1 (en) 2020-06-08
MA49278A (fr) 2020-04-08
CO2019013317A2 (es) 2020-01-17
MX2019014187A (es) 2020-01-27
PE20191845A1 (es) 2019-12-31
RU2019143004A (ru) 2021-07-09
WO2018220034A1 (en) 2018-12-06
US20200157546A1 (en) 2020-05-21
US20220042022A1 (en) 2022-02-10
CA3062590A1 (en) 2018-12-06
AU2018277219A1 (en) 2019-12-19
IL271039A (en) 2020-01-30
JP2020521491A (ja) 2020-07-27
US20190055564A1 (en) 2019-02-21
CL2019003337A1 (es) 2020-05-15

Similar Documents

Publication Publication Date Title
JP7416852B2 (ja) Htra1の発現を調節するためのアンチセンスオリゴヌクレオチド
KR20210121199A (ko) 올리고뉴클레오티드 조성물 및 이의 방법
KR20190040098A (ko) 안티센스 핵산
US20220042022A1 (en) Antisense oligonucleotides for modulating htra1 expression
EP3980539A1 (en) Antisense oligonucleotides targeting atxn3
BR112020016170A2 (pt) Oligonucleotídeos terapêutico e antissenso, conjugado, sal farmaceuticamente aceitável, composição farmacêutica, método in vitro ou in vivo para modular a expressão de tmem106b, método para tratar ou prevenir uma doença, uso do oligonucleotídeo e uso ou método
CN111512160B (zh) Htra1 rna拮抗剂的伴随诊断
JP7236707B2 (ja) リピート不安定性と関連する疾患を治療する方法
NZ749395A (en) Antisense oligonucleotides for modulating htra1 expression
HK40021168A (en) Antisense oligonucleotides for modulating htra1 expression
HK1262526A1 (en) Antisense oligonucleotides for modulating htra1 expression
HK1262526B (zh) 用於调节htra1表达的反义寡核苷酸
HK40031713B (zh) Htra1 rna拮抗剂的伴随诊断
HK40031713A (en) Companion diagnostic for htra1 rna antagonists

Legal Events

Date Code Title Description
B11A Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing
B350 Update of information on the portal [chapter 15.35 patent gazette]
B11Y Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette]