IL231983B - Dna containing tricyclo-phosphorothioate - Google Patents

Dna containing tricyclo-phosphorothioate

Info

Publication number
IL231983B
IL231983B IL231983A IL23198314A IL231983B IL 231983 B IL231983 B IL 231983B IL 231983 A IL231983 A IL 231983A IL 23198314 A IL23198314 A IL 23198314A IL 231983 B IL231983 B IL 231983B
Authority
IL
Israel
Prior art keywords
tricyclo
phosphorothioate dna
phosphorothioate
dna
Prior art date
Application number
IL231983A
Other languages
English (en)
Hebrew (he)
Other versions
IL231983A0 (en
Original Assignee
Inst Nat Sante Rech Med
Centre Nat Rech Scient
Univ Pierre Et Marie Curie Paris 6
Univ Bern
Association Inst De Myologie
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 Inst Nat Sante Rech Med, Centre Nat Rech Scient, Univ Pierre Et Marie Curie Paris 6, Univ Bern, Association Inst De Myologie filed Critical Inst Nat Sante Rech Med
Publication of IL231983A0 publication Critical patent/IL231983A0/en
Publication of IL231983B publication Critical patent/IL231983B/en

Links

Classifications

    • 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)
IL231983A 2011-10-13 2014-04-07 Dna containing tricyclo-phosphorothioate IL231983B (en)

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 (2)

Publication Number Publication Date
IL231983A0 IL231983A0 (en) 2014-05-28
IL231983B true IL231983B (en) 2018-04-30

Family

ID=44799824

Family Applications (1)

Application Number Title Priority Date Filing Date
IL231983A IL231983B (en) 2011-10-13 2014-04-07 Dna containing tricyclo-phosphorothioate

Country Status (14)

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

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EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
EP2762567B1 (en) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
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
BR122020016865B1 (pt) 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
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EP3480312A1 (en) 2013-09-11 2019-05-08 Synthena AG Nucleic acids and methods for the treatment of pompe disease
ES2806087T3 (es) 2014-07-31 2021-02-16 Association Inst De Myologie Tratamiento de la esclerosis lateral amiotrófica
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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US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
WO2017050836A1 (en) 2015-09-21 2017-03-30 Association Institut De Myologie Antisense oligonucleotides and uses thereof
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
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
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
KR102329187B1 (ko) 2016-04-29 2021-11-22 사렙타 쎄러퓨틱스, 인코퍼레이티드 인간 lmna를 표적화하는 올리고뉴클레오타이드 유사체
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
DK3464305T3 (da) 2016-05-24 2024-10-28 Sarepta Therapeutics Inc Fremgangsmåder til fremstilling af oligomerer
AU2017270975B2 (en) 2016-05-24 2021-04-15 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
PT3464306T (pt) 2016-05-24 2024-05-17 Sarepta Therapeutics Inc Processos para preparar oligómeros morfolino de fosforodiamidato
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
SG11201810143PA (en) 2016-06-30 2019-01-30 Sarepta Therapeutics Inc Exon skipping oligomers for muscular dystrophy
CA3037663A1 (en) 2016-09-23 2018-03-29 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
PL3554552T3 (pl) 2016-12-19 2022-11-21 Sarepta Therapeutics, Inc. Koniugaty oligomerów do pomijania egzonów dla dystrofii mięśniowej
SMT202200366T1 (it) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
MY205600A (en) 2016-12-19 2024-10-29 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
US11767520B2 (en) 2017-04-20 2023-09-26 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
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IL318474A (en) 2018-12-13 2025-03-01 Sarepta Therapeutics Inc Axon-skipping oligomer complexes for muscular dystrophy
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JP2024539223A (ja) 2021-10-22 2024-10-28 サレプタ セラピューティクス, インコーポレイテッド 末梢ミエリンタンパク質22関連疾患の処置のためのモルフォリノオリゴマー
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Also Published As

Publication number Publication date
CN104245935B (zh) 2018-05-08
JP2015501144A (ja) 2015-01-15
AU2012322903B2 (en) 2017-09-14
BR112014009066B1 (pt) 2020-08-25
WO2013053928A1 (en) 2013-04-18
EP2581448B1 (en) 2015-01-28
CN104245935A (zh) 2014-12-24
DK2581448T3 (en) 2015-04-27
EP2766479B1 (en) 2017-09-13
HK1186205A1 (en) 2014-03-07
ES2535654T3 (es) 2015-05-13
CA2776651A1 (en) 2013-04-13
CA2851970A1 (en) 2013-04-18
BR112014009066B8 (pt) 2021-02-23
US20140296323A1 (en) 2014-10-02
PT2581448E (pt) 2015-05-21
US9738891B2 (en) 2017-08-22
IN2014DN03463A (OSRAM) 2015-06-05
BR112014009066A2 (pt) 2017-04-18
JP6181653B2 (ja) 2017-08-16
BR112014009066A8 (pt) 2018-01-09
ES2651216T3 (es) 2018-01-25
EP2581448A1 (en) 2013-04-17
PL2581448T3 (pl) 2015-08-31
CA2776651C (en) 2021-06-08
AU2012322903A1 (en) 2014-05-29
IL231983A0 (en) 2014-05-28
EP2766479A1 (en) 2014-08-20

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