[go: up one dir, main page]

PE20191845A1 - Oligonucleotidos antisentido para modulacion de la expresion de htra1 - Google Patents

Oligonucleotidos antisentido para modulacion de la expresion de htra1

Info

Publication number
PE20191845A1
PE20191845A1 PE2019002473A PE2019002473A PE20191845A1 PE 20191845 A1 PE20191845 A1 PE 20191845A1 PE 2019002473 A PE2019002473 A PE 2019002473A PE 2019002473 A PE2019002473 A PE 2019002473A PE 20191845 A1 PE20191845 A1 PE 20191845A1
Authority
PE
Peru
Prior art keywords
modulation
htra1
sense oligonucleotides
htra1 expression
expression
Prior art date
Application number
PE2019002473A
Other languages
English (en)
Inventor
Sanchez Ruben Alvarez
Roberto Iacone
Peter Hagedorn
Susanne Kammler
Søren Ottosen
Sindri Traustason
Heidi Rye Hudlebush
Lykke Pedersen
Marco Barrera
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 PE20191845A1 publication Critical patent/PE20191845A1/es

Links

Classifications

    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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)
    • C12Y304/21108—HtrA2 peptidase (3.4.21.108)
    • 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
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/33—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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special 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)

Abstract

La presente invencion se refiere a oligonucleotidos (oligomeros) antisentido que son complementarios a HTRA1, que conducen a la modulacion de la expresion de HTRA1. La modulacion de la expresion de HTRA1 resulta beneficiosa para un abanico de trastornos medicos, tales como la degeneracion macular, por ejemplo, la degeneracion macular asociada a la edad
PE2019002473A 2017-06-01 2018-05-30 Oligonucleotidos antisentido para modulacion de la expresion de htra1 PE20191845A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP17173964 2017-06-01
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
PE20191845A1 true PE20191845A1 (es) 2019-12-31

Family

ID=62245334

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019002473A PE20191845A1 (es) 2017-06-01 2018-05-30 Oligonucleotidos antisentido para modulacion de la expresion de htra1

Country Status (19)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3478839A1 (en) 2016-07-01 2019-05-08 H. Hoffnabb-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis 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
JPWO2022092326A1 (es) * 2020-10-30 2022-05-05

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
EP2341058A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
AU5290499A (en) * 1998-08-03 2000-02-28 Novartis Ag Human htra serine protease
TR200102328T2 (tr) 1999-02-12 2002-01-21 Sankyo Company Limited Yeni nükleosid ve oligonükleotid analogları
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
CA2994089A1 (en) 2002-11-18 2004-06-03 Roche Innovation Center Copenhagen A/S Antisense gapmer oligonucleotides
ATE538798T1 (de) * 2005-05-24 2012-01-15 Isis Pharmaceuticals Inc Zusammensetzungen und ihre verwendungen gegen niedermolekulare ptpase
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
DK2314594T3 (da) 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modificerede bicykliske nukleinsyreanaloger
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
EP2044223B1 (en) * 2006-07-26 2012-06-27 Yale University, Inc. 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
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
TW200911290A (en) * 2007-07-02 2009-03-16 Alcon Res Ltd RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
CA2704787A1 (en) * 2007-11-01 2009-05-07 University Of Iowa Research Foundation Genes and polymorphisms associated with amd
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2011017521A2 (en) 2009-08-06 2011-02-10 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
US9751909B2 (en) 2011-09-07 2017-09-05 Marina Biotech, Inc. Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
WO2013154798A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
JP2016522674A (ja) * 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
HRP20190826T1 (hr) 2012-11-15 2019-06-28 Roche Innovation Center Copenhagen A/S Konjugati oligonukleotida
WO2018087200A1 (en) * 2016-11-11 2018-05-17 Roche Innovation Center Copenhagen A/S Therapeutic oligonucleotides capture and detection

Also Published As

Publication number Publication date
MA49278A (fr) 2020-04-08
JP2020521491A (ja) 2020-07-27
US20220042022A1 (en) 2022-02-10
CL2019003337A1 (es) 2020-05-15
BR112019025290A2 (pt) 2020-06-23
CA3062590A1 (en) 2018-12-06
MX2019014187A (es) 2020-01-27
US20190055564A1 (en) 2019-02-21
CR20190543A (es) 2020-02-10
CN110691849A (zh) 2020-01-14
KR20200015608A (ko) 2020-02-12
TW201907008A (zh) 2019-02-16
IL271039A (en) 2020-01-30
AU2018277219A1 (en) 2019-12-19
CO2019013317A2 (es) 2020-01-17
WO2018220034A1 (en) 2018-12-06
PH12019502636A1 (en) 2020-06-08
US20200157546A1 (en) 2020-05-21
EP3630976A1 (en) 2020-04-08
JP7169995B2 (ja) 2022-11-11
RU2019143004A (ru) 2021-07-09

Similar Documents

Publication Publication Date Title
CO2018013240A2 (es) Oligonucleótidos antisentido para modular la expresión de htra1
BR112017017396A2 (pt) derivados substituídos de nucleosídeos substituídos úteis como agentes anticancerígenos
CO2019013317A2 (es) Oligonucleótidos antisentido para la modulación de la expresión de htra1
BR112017012272A2 (pt) derivados de 2-anilinopirimidina como moduladores de egfr
CL2020000085A1 (es) Moduladores de nlrp3.
UY36126A (es) ?compuestos de 6,8-dioxabiciclo[3.2.1]octano-2,3-diol sustituidos como agentes de direccionamiento al asgpr?.
DOP2016000194A (es) 2-amino-3,5,5-trifluoro-3,4,5,6-tetrahidropiridinas como inhibidores de bace1 para el tratamiento de la enfermedad de alzheimer
CL2016003179A1 (es) El control efectivo y eficaz del fosfato sérico para una osificación óptima
CO2017005959A2 (es) 4-oxo-3,4-dihidro-1,2,3-benzotriazinas como moduladores de gpr139
MX373283B (es) Composiciones y metodos para modular la expresion del receptor de la hormona del crecimiento.
MX2022013454A (es) Moduladores de la actividad del complemento.
NI201700056A (es) 2-amino-3,5-difluoro-6-metil-6-fenil-3,4,5,6-tetrahidropiridinas en calidad de inhibidores de bace1 para el tratamiento de la enfermedad de alzhéimer
BR112016029994A2 (pt) métodos de preparação de análogos de nucleotídeo substituídos
CO2019012767A2 (es) Compuesto de fenil-2-hidroxi-acetilamino-2-metil-fenilo
NI201700055A (es) 2-amino-6-(difluorometil)-5,5-difluoro-6-fenil-3,4,5,6-tetrahidropiridinas como inhibidores de bace1
CR20170247A (es) 2-amino-5,5-difluoro-6-(fluorometil)-6-fenil-3,4,5,6-tetrahidropiridinas como inhibidores de bace1
DOP2016000211A (es) Pirazinas moduladoras de gpr6
CL2020002038A1 (es) Oligonucleótidos para modular la expresión de tmem106b.
MX2019000982A (es) Compuestos y composiciones y usos de los mismos.
AR112908A1 (es) Oligonucleótidos antisentido para modular la expresión de htra1
DOP2017000196A (es) Derivados de tetrahidropiranil benzamida
PE20150620A1 (es) Moleculas de sirna que interfieren con la expresion del receptor adrenergico beta 2 (adrb2)