BR112015024764A2 - oligonucleotídeos de tgf-beta modificados - Google Patents

oligonucleotídeos de tgf-beta modificados

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Publication number
BR112015024764A2
BR112015024764A2 BR112015024764A BR112015024764A BR112015024764A2 BR 112015024764 A2 BR112015024764 A2 BR 112015024764A2 BR 112015024764 A BR112015024764 A BR 112015024764A BR 112015024764 A BR112015024764 A BR 112015024764A BR 112015024764 A2 BR112015024764 A2 BR 112015024764A2
Authority
BR
Brazil
Prior art keywords
tgf
nucleic acid
acid sequence
oligonucleotide
sequence
Prior art date
Application number
BR112015024764A
Other languages
English (en)
Other versions
BR112015024764B1 (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 BR112015024764A2 publication Critical patent/BR112015024764A2/pt
Publication of BR112015024764B1 publication Critical patent/BR112015024764B1/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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/06Antipsoriatics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • 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
    • C12N2310/32Chemical 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
    • C12N2310/34Spatial 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (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 selected regions are preferably the region of nucleic acid no. 1380 to 1510, no. 1660 to 1680, no. 2390 to 2410, or no. 2740 to 2810 of the tgf-beta2 nucleic acid sequence of seq id no. 1, specific regions of the tgf-beta1 nucleic acid sequence of seq id no. 149, or specific regions of the tgf-beta3 nucleic acid sequence of seq id no. 267. the invention further relates to pharmaceutical compositions comprising such oligonucleotide, wherein the composition or the oligonucleotide is used in the prevention and/or treatment of a malignant and/or benign tumor, an immunologic disease, fibrosis, glaucoma, etc.. tradução resumo resumo patente de invenção: "oligonucleotídeos de tgf-beta modificados". a invenção refere-se a um oligonucleotídeo consistindo em 10 a 20 nucleotídeos de regiões selecionadas da sequência ade ácidos nucleicos de tgf-beta1, tgf-beta2 ou tgf-beta3, que compreende nucleotídeos modificados tais como nucleotídeos lna, ena, polialquileno óxido-, 2'-flúor, 2'-o-metóxi e/ou 2'-o-metil modificados. as regiões selecionadas são de preferência a região de ácido nucleico n° 1380 a 1510, n° 1660 a 1680, n° 2390 a 2410, ou n° 2740 a 2810 da sequência de ácidos nucleicos de tgf-beta2 de seq id n° 1, regiões específicas da sequência de ácidos nucleicos de tgf-beta1 de seq id n° 149, ou regiões específicas da sequência de ácidos nucleicos de seq id n° 267. a invenção refere-se ainda a composições farmacêuticas compreendendo tal oligonucleotídeo, onde a composição ou o oligonucleotídeo é usado na prevenção e/ou no tratamento de um tumor maligno e/ou benigno, um doença imunológica, fibrose, glaucoma, etc.
BR112015024764-4A 2013-03-27 2014-03-27 Oligonucleotídeos de tgf-beta modificados, seu uso e composições farmacêuticas que os compreendem BR112015024764B1 (pt)

Applications Claiming Priority (7)

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
EP13199826 2013-12-30
EP2014154835 2014-10-02

Publications (2)

Publication Number Publication Date
BR112015024764A2 true BR112015024764A2 (pt) 2017-10-24
BR112015024764B1 BR112015024764B1 (pt) 2022-06-28

Family

ID=50390096

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015024764-4A BR112015024764B1 (pt) 2013-03-27 2014-03-27 Oligonucleotídeos de tgf-beta modificados, seu uso e composições farmacêuticas que os compreendem

Country Status (14)

Country Link
US (2) US9926563B2 (pt)
EP (1) EP2978845B8 (pt)
JP (1) JP6492053B2 (pt)
KR (1) KR102108599B1 (pt)
CN (2) CN105378083B (pt)
BR (1) BR112015024764B1 (pt)
CA (1) CA2908096C (pt)
DK (1) DK2978845T3 (pt)
EA (1) EA201591590A1 (pt)
ES (1) ES2808862T3 (pt)
HK (1) HK1221253A1 (pt)
PL (1) PL2978845T3 (pt)
PT (1) PT2978845T (pt)
WO (1) WO2014154835A2 (pt)

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KR20180103816A (ko) 2016-02-09 2018-09-19 오토텔릭 엘엘씨 췌장암을 치료하기 위한 조성물과 방법
MA45469A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45471A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de phosphatidylinositol-3-kinase et leurs utilisations
MA45340A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques du récepteur des androgènes et leurs utilisations
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45470A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45349A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45468A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques myc et utilisations
WO2018087359A1 (en) * 2016-11-11 2018-05-17 Isarna Therapeutics Gmbh Tgf-beta oligonucleotide for use in treatment of ophthalmic diseases
KR20240007965A (ko) 2017-12-06 2024-01-17 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
EP4121063A4 (en) 2020-03-19 2024-07-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS FOR TREATING FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
KR20220039639A (ko) * 2020-09-21 2022-03-29 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
CN113073082A (zh) * 2021-03-19 2021-07-06 广州远想生物科技有限公司 TGF-β3间充质干细胞外泌体及其制备方法和应用
WO2023043953A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
CN118251490A (zh) * 2021-11-01 2024-06-25 奥托泰利克生物公司 反义寡核苷酸

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Also Published As

Publication number Publication date
WO2014154835A3 (en) 2014-11-20
KR102108599B1 (ko) 2020-05-08
CN109234274B (zh) 2021-09-21
PT2978845T (pt) 2020-08-03
CA2908096A1 (en) 2014-10-02
EP2978845A2 (en) 2016-02-03
HK1221253A1 (zh) 2017-05-26
CN105378083B (zh) 2018-09-21
WO2014154835A2 (en) 2014-10-02
CN109234274A (zh) 2019-01-18
US20160076037A1 (en) 2016-03-17
CN105378083A (zh) 2016-03-02
US10538768B2 (en) 2020-01-21
EP2978845B8 (en) 2020-06-17
KR20150140701A (ko) 2015-12-16
EA201591590A1 (ru) 2016-05-31
US20180230473A1 (en) 2018-08-16
BR112015024764B1 (pt) 2022-06-28
JP6492053B2 (ja) 2019-03-27
DK2978845T3 (da) 2020-08-10
ES2808862T3 (es) 2021-03-02
US9926563B2 (en) 2018-03-27
CA2908096C (en) 2022-05-03
JP2016515385A (ja) 2016-05-30
PL2978845T3 (pl) 2020-11-16
EP2978845B1 (en) 2020-04-29

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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]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
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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