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)
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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pain & Pain Management (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 (pl)
EP (2) EP2581448B1 (pl)
JP (1) JP6181653B2 (pl)
CN (1) CN104245935B (pl)
AU (1) AU2012322903B2 (pl)
BR (1) BR112014009066B8 (pl)
CA (2) CA2776651C (pl)
DK (1) DK2581448T3 (pl)
ES (2) ES2535654T3 (pl)
IL (1) IL231983B (pl)
IN (1) IN2014DN03463A (pl)
PL (1) PL2581448T3 (pl)
PT (1) PT2581448E (pl)
WO (1) WO2013053928A1 (pl)

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KR102239374B1 (ko) 2009-11-12 2021-04-14 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
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EP3662912B1 (en) 2013-03-15 2025-12-03 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
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US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
EP3353301A1 (en) 2015-09-21 2018-08-01 Association Institut de Myologie Antisense oligonucleotides and uses thereof
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
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
BR112018071477A2 (pt) 2016-04-18 2019-02-19 Sarepta Therapeutics, Inc. oligômeros antisenso e métodos de utilização dos mesmos para tratamento de doenças associadas com o gene da alfa-glicosidase ácida
MX2018013157A (es) 2016-04-29 2019-05-23 Sarepta Therapeutics Inc Analogos de oligonucleotidos dirigidos a lmna humana.
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
HRP20240583T1 (hr) 2016-05-24 2024-07-19 Sarepta Therapeutics, Inc. Postupci za pripremu fosforodiamidat morfolino oligomera
US10947533B2 (en) 2016-05-24 2021-03-16 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
SG10202101836TA (en) 2016-05-24 2021-03-30 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
EP3464305B1 (en) 2016-05-24 2024-08-21 Sarepta Therapeutics, Inc. Processes for preparing oligomers
TW201811807A (zh) 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
WO2018055577A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
EP3516061A1 (en) 2016-09-23 2019-07-31 Synthena AG Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
LT3554553T (lt) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
PL3554554T3 (pl) 2016-12-19 2022-12-19 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
RS63621B1 (sr) 2016-12-19 2022-10-31 Sarepta Therapeutics Inc Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju
AU2018256435B2 (en) 2017-04-20 2025-03-13 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
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US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
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TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
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Also Published As

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

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