PL2581448T3 - Tricyklo-tiofosforanowy DNA - Google Patents

Tricyklo-tiofosforanowy DNA

Info

Publication number
PL2581448T3
PL2581448T3 PL11185129T PL11185129T PL2581448T3 PL 2581448 T3 PL2581448 T3 PL 2581448T3 PL 11185129 T PL11185129 T PL 11185129T PL 11185129 T PL11185129 T PL 11185129T PL 2581448 T3 PL2581448 T3 PL 2581448T3
Authority
PL
Poland
Prior art keywords
tricyclo
phosphorothioate dna
phosphorothioate
dna
Prior art date
Application number
PL11185129T
Other languages
English (en)
Polish (pl)
Inventor
Christian Leumann
Luis Garcia
Thomas Voit
Original Assignee
Association Inst De Myologie
Univ Bern
Univ Pierre Et Marie Curie Paris 6
Inst Nat Sante Rech Med
Centre Nat Rech Scient
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 Association Inst De Myologie, Univ Bern, Univ Pierre Et Marie Curie Paris 6, Inst Nat Sante Rech Med, Centre Nat Rech Scient filed Critical Association Inst De Myologie
Publication of PL2581448T3 publication Critical patent/PL2581448T3/pl

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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
    • 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
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
PL11185129T 2011-10-13 2011-10-13 Tricyklo-tiofosforanowy DNA PL2581448T3 (pl)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA

Publications (1)

Publication Number Publication Date
PL2581448T3 true PL2581448T3 (pl) 2015-08-31

Family

ID=44799824

Family Applications (1)

Application Number Title Priority Date Filing Date
PL11185129T PL2581448T3 (pl) 2011-10-13 2011-10-13 Tricyklo-tiofosforanowy DNA

Country Status (14)

Country Link
US (1) US9738891B2 (enExample)
EP (2) EP2581448B1 (enExample)
JP (1) JP6181653B2 (enExample)
CN (1) CN104245935B (enExample)
AU (1) AU2012322903B2 (enExample)
BR (1) BR112014009066B8 (enExample)
CA (2) CA2776651C (enExample)
DK (1) DK2581448T3 (enExample)
ES (2) ES2535654T3 (enExample)
IL (1) IL231983B (enExample)
IN (1) IN2014DN03463A (enExample)
PL (1) PL2581448T3 (enExample)
PT (1) PT2581448E (enExample)
WO (1) WO2013053928A1 (enExample)

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IL280443B (en) 2013-03-14 2022-07-01 Sarepta Therapeutics Inc Preparations that skip axon for the treatment of muscular dystrophy
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WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
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SG11201809468XA (en) 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
KR102504759B1 (ko) 2016-05-24 2023-02-28 사렙타 쎄러퓨틱스 인코퍼레이티드 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법
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MD3464305T2 (ro) 2016-05-24 2025-01-31 Sarepta Therapeutics Inc Procedee de preparare de oligomeri
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
SG10202101836TA (en) 2016-05-24 2021-03-30 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
CN109311919A (zh) 2016-06-30 2019-02-05 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体
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MD3554553T2 (ro) 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară
MA65895B1 (fr) 2016-12-19 2024-06-28 Sarepta Therapeutics, Inc. Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire
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CN110945005A (zh) * 2017-04-20 2020-03-31 新特纳股份公司 包含三环dna核苷的修饰的寡聚化合物及其用途
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BR112022020412A2 (pt) 2020-04-09 2023-05-02 Association Inst De Myologie Sequências antissenso para tratar esclerose lateral amiotrófica
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Also Published As

Publication number Publication date
WO2013053928A1 (en) 2013-04-18
EP2766479B1 (en) 2017-09-13
US9738891B2 (en) 2017-08-22
AU2012322903A1 (en) 2014-05-29
US20140296323A1 (en) 2014-10-02
IL231983B (en) 2018-04-30
BR112014009066B1 (pt) 2020-08-25
ES2651216T3 (es) 2018-01-25
IL231983A0 (en) 2014-05-28
HK1186205A1 (en) 2014-03-07
EP2581448B1 (en) 2015-01-28
DK2581448T3 (en) 2015-04-27
PT2581448E (pt) 2015-05-21
CN104245935A (zh) 2014-12-24
JP6181653B2 (ja) 2017-08-16
EP2581448A1 (en) 2013-04-17
IN2014DN03463A (enExample) 2015-06-05
AU2012322903B2 (en) 2017-09-14
BR112014009066A8 (pt) 2018-01-09
BR112014009066B8 (pt) 2021-02-23
BR112014009066A2 (pt) 2017-04-18
CA2851970A1 (en) 2013-04-18
EP2766479A1 (en) 2014-08-20
ES2535654T3 (es) 2015-05-13
CN104245935B (zh) 2018-05-08
JP2015501144A (ja) 2015-01-15
CA2776651C (en) 2021-06-08
CA2776651A1 (en) 2013-04-13

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