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
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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)
  • 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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
IN3463DEN2014 2011-10-13 2014-04-30 IN2014DN03463A (enExample)

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 (enExample) 2015-06-05

Family

ID=44799824

Family Applications (1)

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

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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ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
EP2762567B1 (en) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
IL282239B2 (en) 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
ES2739850T3 (es) 2013-09-11 2020-02-04 Synthena Ag Acidos nucleicos y procedimientos para el tratamiento de la enfermedad de Pompe
CA2955285C (en) 2014-07-31 2023-09-26 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
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
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
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
NZ747685A (en) 2016-04-29 2023-05-26 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
MX383656B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
SG10202101834QA (en) 2016-05-24 2021-04-29 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
CN109563114B (zh) 2016-05-24 2022-08-12 萨勒普塔医疗公司 用于制备寡聚物的方法
SG11201809497RA (en) 2016-05-24 2018-12-28 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
US20190262375A1 (en) 2016-06-30 2019-08-29 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
US11118179B2 (en) 2016-09-23 2021-09-14 Synthena Ag Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
HRP20240705T1 (hr) 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
JP7801082B2 (ja) * 2017-04-20 2026-01-16 シンセナ アーゲー トリシクロdnaヌクレオシドを含む改変オリゴマー化合物およびその使用
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EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
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CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
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Also Published As

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

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