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

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TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US11236338B2 (en) 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3044315B1 (en) 2013-09-11 2019-02-20 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
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
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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
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EP3858993A1 (en) 2015-10-09 2021-08-04 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
MX2018013157A (es) 2016-04-29 2019-05-23 Sarepta Therapeutics Inc Analogos de oligonucleotidos dirigidos a lmna humana.
CA3024456A1 (en) 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
BR112018074346B1 (pt) 2016-05-24 2023-01-03 Sarepta Therapeutics, Inc. Composto oligomérico e processo para preparar um composto oligomérico
MD3464306T2 (ro) 2016-05-24 2024-08-31 Sarepta Therapeutics Inc Procedee de preparare a oligomerilor morfolino fosforodiamidați
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
US10947533B2 (en) 2016-05-24 2021-03-16 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
EP3516061A1 (en) 2016-09-23 2019-07-31 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
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR20240006023A (ko) 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
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Also Published As

Publication number Publication date
CA2776651C (en) 2021-06-08
BR112014009066B8 (pt) 2021-02-23
CN104245935A (zh) 2014-12-24
PT2581448E (pt) 2015-05-21
ES2651216T3 (es) 2018-01-25
EP2581448B1 (en) 2015-01-28
EP2581448A1 (en) 2013-04-17
BR112014009066B1 (pt) 2020-08-25
BR112014009066A2 (pt) 2017-04-18
CA2851970A1 (en) 2013-04-18
JP6181653B2 (ja) 2017-08-16
ES2535654T3 (es) 2015-05-13
IN2014DN03463A (2) 2015-06-05
WO2013053928A1 (en) 2013-04-18
JP2015501144A (ja) 2015-01-15
PL2581448T3 (pl) 2015-08-31
DK2581448T3 (en) 2015-04-27
CA2776651A1 (en) 2013-04-13
IL231983A0 (en) 2014-05-28
AU2012322903A1 (en) 2014-05-29
HK1186205A1 (en) 2014-03-07
CN104245935B (zh) 2018-05-08
EP2766479A1 (en) 2014-08-20
US9738891B2 (en) 2017-08-22
BR112014009066A8 (pt) 2018-01-09
US20140296323A1 (en) 2014-10-02
AU2012322903B2 (en) 2017-09-14
EP2766479B1 (en) 2017-09-13

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