IN2014DN03463A - - Google Patents

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Publication number
IN2014DN03463A
IN2014DN03463A IN3463DEN2014A IN2014DN03463A IN 2014DN03463 A IN2014DN03463 A IN 2014DN03463A IN 3463DEN2014 A IN3463DEN2014 A IN 3463DEN2014A IN 2014DN03463 A IN2014DN03463 A IN 2014DN03463A
Authority
IN
India
Prior art keywords
relates
tricyclo
sequence
nucleic acid
acid molecule
Prior art date
Application number
Other languages
English (en)
Inventor
Christian Leumann
Luis Garcia
Thomas Voit
Original Assignee
Ass Inst De Myologie
Universität 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.)
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Application filed by Ass Inst De Myologie, Universität Bern, Univ Pierre Et Marie Curie Paris 6, Inst Nat Sante Rech Med, Centre Nat Rech Scient filed Critical Ass Inst De Myologie
Publication of IN2014DN03463A publication Critical patent/IN2014DN03463A/en

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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)
IN3463DEN2014 2011-10-13 2014-04-30 IN2014DN03463A (enrdf_load_stackoverflow)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161546942P 2011-10-13 2011-10-13
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA
CA2776651A CA2776651C (en) 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna
PCT/EP2012/070349 WO2013053928A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Publications (1)

Publication Number Publication Date
IN2014DN03463A true IN2014DN03463A (enrdf_load_stackoverflow) 2015-06-05

Family

ID=44799824

Family Applications (1)

Application Number Title Priority Date Filing Date
IN3463DEN2014 IN2014DN03463A (enrdf_load_stackoverflow) 2011-10-13 2014-04-30

Country Status (14)

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

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EP3431603A1 (en) 2009-11-12 2019-01-23 The University Of Western Australia Antisense molecules and methods for treating pathologies
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
KR20230116945A (ko) 2013-03-14 2023-08-04 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
AU2014233456B2 (en) 2013-03-15 2019-02-21 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
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WO2015036451A1 (en) 2013-09-11 2015-03-19 Synthena Ag Nucleic acids and methods for the treatment of pompe disease
EP3174981B1 (en) 2014-07-31 2020-03-25 Association Institut de Myologie Treatment of amyotrophic lateral sclerosis
BR112017004056A2 (pt) 2014-09-12 2017-12-05 Biogen Ma Inc composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016196670A1 (en) 2015-06-01 2016-12-08 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
US20190177723A1 (en) 2015-10-09 2019-06-13 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
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IL263044B2 (en) 2016-05-24 2024-06-01 Sarepta Therapeutics Inc Processes for the preparation of phosphorodiamidate morpholino oligomers
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
SI3464306T1 (sl) 2016-05-24 2024-08-30 Sarepta Therapeutics, Inc. Postopki za pripravo fosforodiamidatnih morfolino oligomerov
FI3464305T3 (fi) 2016-05-24 2024-10-30 Sarepta Therapeutics Inc Menetelmiä oligomeerien valmistamiseksi
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
JP2019525742A (ja) 2016-06-30 2019-09-12 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
US11118179B2 (en) 2016-09-23 2021-09-14 Synthena Ag Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
CN110636866B (zh) 2016-12-19 2024-10-18 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
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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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Also Published As

Publication number Publication date
AU2012322903A1 (en) 2014-05-29
IL231983A0 (en) 2014-05-28
ES2535654T3 (es) 2015-05-13
JP2015501144A (ja) 2015-01-15
IL231983B (en) 2018-04-30
PL2581448T3 (pl) 2015-08-31
ES2651216T3 (es) 2018-01-25
CN104245935B (zh) 2018-05-08
BR112014009066A8 (pt) 2018-01-09
US9738891B2 (en) 2017-08-22
CA2776651C (en) 2021-06-08
DK2581448T3 (en) 2015-04-27
CN104245935A (zh) 2014-12-24
EP2581448B1 (en) 2015-01-28
EP2766479A1 (en) 2014-08-20
EP2766479B1 (en) 2017-09-13
PT2581448E (pt) 2015-05-21
BR112014009066A2 (pt) 2017-04-18
AU2012322903B2 (en) 2017-09-14
JP6181653B2 (ja) 2017-08-16
CA2776651A1 (en) 2013-04-13
CA2851970A1 (en) 2013-04-18
EP2581448A1 (en) 2013-04-17
BR112014009066B8 (pt) 2021-02-23
US20140296323A1 (en) 2014-10-02
WO2013053928A1 (en) 2013-04-18
BR112014009066B1 (pt) 2020-08-25
HK1186205A1 (en) 2014-03-07

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