BR112015024760A2 - oligonucleotídeo beta modificado para uso em um método de prevenção e/ou tratamento de uma doença oftálmica - Google Patents

oligonucleotídeo beta modificado para uso em um método de prevenção e/ou tratamento de uma doença oftálmica

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Publication number
BR112015024760A2
BR112015024760A2 BR112015024760A BR112015024760A BR112015024760A2 BR 112015024760 A2 BR112015024760 A2 BR 112015024760A2 BR 112015024760 A BR112015024760 A BR 112015024760A BR 112015024760 A BR112015024760 A BR 112015024760A BR 112015024760 A2 BR112015024760 A2 BR 112015024760A2
Authority
BR
Brazil
Prior art keywords
tgf
oligonucleotide
macular degeneration
nucleotides
modified
Prior art date
Application number
BR112015024760A
Other languages
English (en)
Other versions
BR112015024760B1 (pt
BR112015024760B8 (pt
Inventor
Uhlmann Eugen
Jaschinski Frank
Janicot Michel
Original Assignee
Isarna Therapeutics Gmbh
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 Isarna Therapeutics Gmbh filed Critical Isarna Therapeutics Gmbh
Publication of BR112015024760A2 publication Critical patent/BR112015024760A2/pt
Publication of BR112015024760B1 publication Critical patent/BR112015024760B1/pt
Publication of BR112015024760B8 publication Critical patent/BR112015024760B8/pt

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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
    • C12N15/1136Non-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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • A61P27/06Antiglaucoma agents or miotics
    • 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
    • A61P27/12Ophthalmic agents for cataracts
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

abstract the invention refers to an oligonucleotide consisting of 10 to 20 nucleotides of selected regions of the tgf-beta1, tgf-beta2 or tgf-beta3 nucleic acid sequence, which comprises modified nucleotides such as lna, ena, polyalkylene oxide-, 2'-fluoro, 2'-o- methoxy and/or 2'-o-methyl modified nucleotides. the invention further relates to pharmaceutical compositions comprising such oligonucleotide, wherein the composition or the oligonucleotide is used in a method for the prevention and/or treatment of glaucoma, posterior capsular opacification, dry eye, marfan or loeys- dietz syndrome, riboblastoma, choroidcarcinoma, macular degeneration, such as age- related macular degeneration, diabetic macular endma, or cataract. ------------------------------------------------------------------------------------------ tradução do resumo resumo patente de invenção: "oligonucleotídeo beta modificado para uso em um método de prevenção e/ou tratamen-to de uma doença oftálmica". a invenção se refere a um oligonucleotídeo consistindo de 10 a 20 nucleotídeos de regiões selecionadas da sequência de nucleo-tídeo de tgf-beta1, tgf-beta2 ou tgf-beta3, que compreende nu-cleotídeo modificados, tais como nucleotídeos modificados de lna, ena, óxido de polialquileno-, 2'-fluoro, 2'-o-meóxi e/ou 2'-o-metil. a invenção se relaciona adicionalmente a composições farmacêuticas compreendendo tal oligonucleotídeo, no qual a composição ou o oligo-nucleotídeo é usado em um método para a prevenção e/ou tratamento de glaucoma, opacificação capsular posterior, olho seco, síndrome de marfan ou de loeys-dietz, riboblastoma, coroidocarcinoma, degenera-ção macular, tal como degeneração macular relacionada à idade, edema macular diabético, ou catarata.
BR112015024760A 2013-03-27 2014-03-27 Oligonucleotídeo antissenso, composição farmacêutica e uso dos mesmos na prevenção e/ou tratamento de uma doença oftálmica BR112015024760B8 (pt)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
EP13161474 2013-03-27
EP13161474.5 2013-03-27
EP13173078.0 2013-06-20
EP13173078 2013-06-20
EP13199826.2 2013-12-30
EP13199831.2 2013-12-30
EP13199826 2013-12-30
EP13199831 2013-12-30
EP13199838.7 2013-12-30
EP13199838 2013-12-30
PCT/EP2014/056222 WO2014154836A2 (en) 2013-03-27 2014-03-27 Modified tgf-beta oligonucleotide for use in a method of preventing and/or treating an ophthalmic disease

Publications (3)

Publication Number Publication Date
BR112015024760A2 true BR112015024760A2 (pt) 2017-10-24
BR112015024760B1 BR112015024760B1 (pt) 2021-09-21
BR112015024760B8 BR112015024760B8 (pt) 2022-06-21

Family

ID=50440642

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015024760A BR112015024760B8 (pt) 2013-03-27 2014-03-27 Oligonucleotídeo antissenso, composição farmacêutica e uso dos mesmos na prevenção e/ou tratamento de uma doença oftálmica

Country Status (17)

Country Link
US (3) US9688988B2 (pt)
EP (1) EP2978846B1 (pt)
JP (1) JP6475226B2 (pt)
KR (1) KR102094572B1 (pt)
CN (1) CN105283551B (pt)
BR (1) BR112015024760B8 (pt)
CA (1) CA2908101C (pt)
DK (1) DK2978846T3 (pt)
EA (1) EA201591594A1 (pt)
ES (1) ES2784213T3 (pt)
HK (1) HK1221254A1 (pt)
HU (1) HUE049246T2 (pt)
LT (1) LT2978846T (pt)
PL (1) PL2978846T3 (pt)
PT (1) PT2978846T (pt)
SI (1) SI2978846T1 (pt)
WO (1) WO2014154836A2 (pt)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018009895A1 (en) * 2016-07-08 2018-01-11 Autotelic Llc Methods for trabedersen dosing by auc
WO2018087359A1 (en) * 2016-11-11 2018-05-17 Isarna Therapeutics Gmbh Tgf-beta oligonucleotide for use in treatment of ophthalmic diseases
US11771703B2 (en) 2017-03-17 2023-10-03 The Johns Hopkins University Targeted epigenetic therapy against distal regulatory element of TGFβ2 expression
CN113018508A (zh) * 2021-03-15 2021-06-25 西安交通大学医学院第一附属医院 一种表面修饰的人工晶状体及其制备方法
KR102549208B1 (ko) * 2021-04-08 2023-06-30 연세대학교 산학협력단 심혈관 질환과 관련된 LncRNA 및 이의 용도

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994025588A2 (en) 1993-04-30 1994-11-10 Biognostik Gesellschaft für Biomolekulare Diagnostik mbH ANTISENSE-OLIGONUCLEOTIDES FOR THE TREATMENT OF IMMUNOSUPPRESSIVE EFFECTS OF TRANSFORMING GROWTH FACTOR-β (TGF-β)
EP1008649B1 (en) 1993-04-30 2003-03-26 Biognostik Gesellschaft für biomolekulare Diagnostik mbH Antisense-oligonucleotides for the treatment of immuno-suppressive effects of transforming growth factor-b2(TGF-b2)
US20040006030A1 (en) 2002-07-02 2004-01-08 Isis Pharmaceuticals Inc. Antisense modulation of TGF-beta 2 expression
EP1568383A3 (en) 2004-02-27 2005-11-16 Antisense Pharma GmbH Use of an oligonucleotide or its active derivative for the preparation of a pharmaceutical composition for inhibiting the formation of metastases in cancer treatment
JP5650367B2 (ja) * 2004-02-27 2015-01-07 アンティセンス ファルマ ゲゼルシャフト ミット ベシュレンクテル ハフツング 医薬組成物
EP1935428A1 (en) 2006-12-22 2008-06-25 Antisense Pharma GmbH Oligonucleotide-polymer conjugates
JP2013531981A (ja) * 2010-06-11 2013-08-15 アンチセンス・ファーマ・ゲーエムベーハー 選択的オリゴヌクレオチド修飾の方法
EP2453017A1 (en) * 2010-11-12 2012-05-16 Antisense Pharma GmbH Oligonucleotides for use in prophylaxis and/or treatment of TGF-beta1 and TGF-beta2, TGF-beta2 and TGF-beta3, TGF-beta1 and TGF-beta3, or TGF-beta1, TGF-beta2, and TGF-beta3 mRNA overexpressing diseases
US10058623B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating UTRN expression

Also Published As

Publication number Publication date
KR102094572B1 (ko) 2020-03-30
US9688988B2 (en) 2017-06-27
US10125368B2 (en) 2018-11-13
PL2978846T4 (pl) 2020-09-21
EA201591594A1 (ru) 2016-05-31
CA2908101A1 (en) 2014-10-02
EP2978846B1 (en) 2019-12-25
EP2978846A2 (en) 2016-02-03
BR112015024760B1 (pt) 2021-09-21
JP6475226B2 (ja) 2019-02-27
ES2784213T3 (es) 2020-09-23
HK1221254A1 (zh) 2017-05-26
US20190144865A1 (en) 2019-05-16
LT2978846T (lt) 2020-04-10
SI2978846T1 (sl) 2020-07-31
CN105283551A (zh) 2016-01-27
WO2014154836A2 (en) 2014-10-02
DK2978846T3 (da) 2020-04-06
WO2014154836A3 (en) 2014-11-20
BR112015024760B8 (pt) 2022-06-21
CA2908101C (en) 2021-06-22
HUE049246T2 (hu) 2020-09-28
PT2978846T (pt) 2020-04-21
JP2016519573A (ja) 2016-07-07
US20160040167A1 (en) 2016-02-11
KR20150140702A (ko) 2015-12-16
US20170314025A1 (en) 2017-11-02
PL2978846T3 (pl) 2020-09-21
CN105283551B (zh) 2018-11-06

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B06A Patent application procedure suspended [chapter 6.1 patent gazette]
B15K Others concerning applications: alteration of classification

Free format text: AS CLASSIFICACOES ANTERIORES ERAM: C12N 15/113 , A61K 31/713 , C07H 21/02 , A61P 27/02 , A61P 35/00

Ipc: C12N 15/113 (2010.01), C07H 21/02 (2006.01), A61K

B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

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