PT2581448E - Dna triciclo-fosforotioato - Google Patents

Dna triciclo-fosforotioato

Info

Publication number
PT2581448E
PT2581448E PT111851291T PT11185129T PT2581448E PT 2581448 E PT2581448 E PT 2581448E PT 111851291 T PT111851291 T PT 111851291T PT 11185129 T PT11185129 T PT 11185129T PT 2581448 E PT2581448 E PT 2581448E
Authority
PT
Portugal
Prior art keywords
phosphorotioate
tricyclo
dna
dna tricyclo
Prior art date
Application number
PT111851291T
Other languages
English (en)
Portuguese (pt)
Inventor
Leumann Christian
Garcia Luis
Voit Thomas
Original Assignee
Institut National De La Santé Et De La Rech Médicale (Inserm)
Universität Bern
Univ Pierre Et Marie Curie Paris 6
Association Inst De Myologie
Centre National De La Recherche Scient (Cnrs)
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 Institut National De La Santé Et De La Rech Médicale (Inserm), Universität Bern, Univ Pierre Et Marie Curie Paris 6, Association Inst De Myologie, Centre National De La Recherche Scient (Cnrs) filed Critical Institut National De La Santé Et De La Rech Médicale (Inserm)
Publication of PT2581448E publication Critical patent/PT2581448E/pt

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)
  • 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)
PT111851291T 2011-10-13 2011-10-13 Dna triciclo-fosforotioato PT2581448E (pt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
PT2581448E true PT2581448E (pt) 2015-05-21

Family

ID=44799824

Family Applications (1)

Application Number Title Priority Date Filing Date
PT111851291T PT2581448E (pt) 2011-10-13 2011-10-13 Dna triciclo-fosforotioato

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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ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
EP4174178A1 (en) 2008-10-24 2023-05-03 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
WO2011057350A1 (en) 2009-11-12 2011-05-19 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
JP6449231B2 (ja) 2013-03-14 2019-01-09 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィを処置するためのエキソンスキッピング組成物
JP2016516066A (ja) 2013-03-15 2016-06-02 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィを処置するための改善された組成物
JP6618910B2 (ja) 2013-09-05 2019-12-11 サレプタ セラピューティクス,インコーポレイテッド 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン2包含
EP3044315B1 (en) 2013-09-11 2019-02-20 Synthena AG Nucleic acids and methods for the treatment of pompe disease
WO2016016449A1 (en) 2014-07-31 2016-02-04 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
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MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
EP3851531A1 (en) 2015-06-01 2021-07-21 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
SG11201808964PA (en) 2016-04-18 2018-11-29 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
MX384727B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros
JP6987081B2 (ja) 2016-05-24 2021-12-22 サレプタ セラピューティクス, インコーポレイテッド ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
IL263044B2 (en) 2016-05-24 2024-06-01 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
TW201811807A (zh) 2016-06-30 2018-04-01 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
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
PT3554552T (pt) 2016-12-19 2022-10-03 Sarepta Therapeutics Inc Conjugados de oligómero de skipping de exão para distrofia muscular
MY195801A (en) 2016-12-19 2023-02-22 Sarepta Therapeutics Inc Exon Skipping Oligomer Conjugates for Muscular Dystrophy
RS63705B1 (sr) 2016-12-19 2022-11-30 Sarepta Therapeutics Inc Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
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
ES2994451T3 (en) 2017-04-20 2025-01-24 Atyr Pharma Inc Compositions for treating lung inflammation
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US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
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TW202449155A (zh) 2018-07-27 2024-12-16 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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TW202517787A (zh) 2020-02-28 2025-05-01 美商Ionis製藥公司 用於調節smn2之化合物及方法
JP7787094B2 (ja) 2020-04-09 2025-12-16 アソシエーション・アンスティトゥート・ドゥ・マイオロジー 筋萎縮性側索硬化症を治療するためのアンチセンス配列
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CA3233242A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
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
JP2025533454A (ja) 2022-09-21 2025-10-07 サレプタ セラピューティクス, インコーポレイテッド Dmdアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率
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Also Published As

Publication number Publication date
IL231983A0 (en) 2014-05-28
ES2535654T3 (es) 2015-05-13
DK2581448T3 (en) 2015-04-27
US9738891B2 (en) 2017-08-22
WO2013053928A1 (en) 2013-04-18
BR112014009066A8 (pt) 2018-01-09
HK1186205A1 (en) 2014-03-07
PL2581448T3 (pl) 2015-08-31
CA2851970A1 (en) 2013-04-18
CN104245935A (zh) 2014-12-24
CA2776651A1 (en) 2013-04-13
EP2581448A1 (en) 2013-04-17
BR112014009066B1 (pt) 2020-08-25
EP2766479B1 (en) 2017-09-13
JP2015501144A (ja) 2015-01-15
JP6181653B2 (ja) 2017-08-16
IL231983B (en) 2018-04-30
EP2766479A1 (en) 2014-08-20
AU2012322903A1 (en) 2014-05-29
CN104245935B (zh) 2018-05-08
CA2776651C (en) 2021-06-08
US20140296323A1 (en) 2014-10-02
ES2651216T3 (es) 2018-01-25
IN2014DN03463A (enExample) 2015-06-05
BR112014009066A2 (pt) 2017-04-18
AU2012322903B2 (en) 2017-09-14
EP2581448B1 (en) 2015-01-28
BR112014009066B8 (pt) 2021-02-23

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