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.)
Filing date
Publication date
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)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (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 (xx)

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 (xx) 2015-06-05

Family

ID=44799824

Family Applications (1)

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

Country Status (15)

Country Link
US (1) US9738891B2 (xx)
EP (2) EP2581448B1 (xx)
JP (1) JP6181653B2 (xx)
CN (1) CN104245935B (xx)
AU (1) AU2012322903B2 (xx)
BR (1) BR112014009066B8 (xx)
CA (2) CA2776651C (xx)
DK (1) DK2581448T3 (xx)
ES (2) ES2535654T3 (xx)
HK (1) HK1186205A1 (xx)
IL (1) IL231983B (xx)
IN (1) IN2014DN03463A (xx)
PL (1) PL2581448T3 (xx)
PT (1) PT2581448E (xx)
WO (1) WO2013053928A1 (xx)

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AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
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PL2499249T3 (pl) 2009-11-12 2019-03-29 Univ Western Australia Cząsteczki antysensowne i sposoby leczenia patologii
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EA202090946A3 (ru) 2013-03-14 2021-07-30 Сарепта Терапьютикс, Инк. Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии
EP3662912A1 (en) 2013-03-15 2020-06-10 Sarepta Therapeutics, Inc. Improved dosages of eteplirsen for treating duchenne muscular dystrophy
CN111235149B (zh) 2013-09-05 2024-04-16 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
US10059947B2 (en) 2013-09-11 2018-08-28 Synthena Ag Nucleic acids and methods for the treatment of Pompe disease
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WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
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MA50829A (fr) 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
CA2999192A1 (en) 2015-09-21 2017-03-30 Association Institut De Myologie Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna 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
EA201892366A1 (ru) 2016-04-18 2019-03-29 Сарепта Терапьютикс, Инк. Антисмысловые олигомеры и способы их применения для лечения заболеваний, связанных с геном кислой альфа-глюкозидазы
EA201892467A1 (ru) 2016-04-29 2019-05-31 Сарепта Терапьютикс, Инк. Олигонуклеотидные аналоги, нацеленные на lmna человека
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45155A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
JP7008642B2 (ja) 2016-05-24 2022-01-25 サレプタ セラピューティクス, インコーポレイテッド オリゴマー調製のためのプロセス
KR20190024977A (ko) 2016-06-30 2019-03-08 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
CA3037663A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified-rna oligonucleotide compositions and uses thereof
MX2019006882A (es) 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
BR112019012651A2 (pt) 2016-12-19 2020-01-28 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
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TW202020153A (zh) 2018-07-27 2020-06-01 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
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Also Published As

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

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