PL2581448T3 - Tricyklo-tiofosforanowy DNA - Google Patents

Tricyklo-tiofosforanowy DNA

Info

Publication number
PL2581448T3
PL2581448T3 PL11185129T PL11185129T PL2581448T3 PL 2581448 T3 PL2581448 T3 PL 2581448T3 PL 11185129 T PL11185129 T PL 11185129T PL 11185129 T PL11185129 T PL 11185129T PL 2581448 T3 PL2581448 T3 PL 2581448T3
Authority
PL
Poland
Prior art keywords
tricyclo
phosphorothioate dna
phosphorothioate
dna
Prior art date
Application number
PL11185129T
Other languages
English (en)
Inventor
Christian Leumann
Luis Garcia
Thomas Voit
Original Assignee
Association Inst De Myologie
Univ 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 Association Inst De Myologie, Univ Bern, Univ Pierre Et Marie Curie Paris 6, Inst Nat Sante Rech Med, Centre Nat Rech Scient filed Critical Association Inst De Myologie
Publication of PL2581448T3 publication Critical patent/PL2581448T3/pl

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (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)
PL11185129T 2011-10-13 2011-10-13 Tricyklo-tiofosforanowy DNA PL2581448T3 (pl)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA

Publications (1)

Publication Number Publication Date
PL2581448T3 true PL2581448T3 (pl) 2015-08-31

Family

ID=44799824

Family Applications (1)

Application Number Title Priority Date Filing Date
PL11185129T PL2581448T3 (pl) 2011-10-13 2011-10-13 Tricyklo-tiofosforanowy DNA

Country Status (15)

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

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EP2500430B1 (en) 2004-06-28 2017-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
CN105838714B (zh) 2009-11-12 2020-07-17 西澳大利亚大学 反义分子和治疗疾病的方法
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
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
ES2739850T3 (es) 2013-09-11 2020-02-04 Synthena Ag Acidos nucleicos y procedimientos para el tratamiento de la enfermedad de Pompe
US10590420B2 (en) 2014-07-31 2020-03-17 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
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
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
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
KR102522059B1 (ko) 2016-04-18 2023-04-14 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법
CA3022303A1 (en) 2016-04-29 2017-11-02 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
CA3024182A1 (en) 2016-05-24 2017-11-30 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
MA45154A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
MX2018016052A (es) 2016-06-30 2019-05-02 Sarepta Therapeutics Inc Oligomeros de omision de exon para distrofia muscular.
US11118179B2 (en) 2016-09-23 2021-09-14 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
EP3554552B1 (en) 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP4122497B1 (en) 2016-12-19 2024-04-10 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
PL3554553T3 (pl) 2016-12-19 2022-11-07 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
CN110536694A (zh) 2017-04-20 2019-12-03 Atyr 医药公司 用于治疗肺部炎症的组合物和方法
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
EP3612546B1 (en) 2017-04-20 2022-07-13 Synthena AG Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
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
WO2020123574A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US20220387624A1 (en) 2019-11-06 2022-12-08 Association Institut De Myologie Combined therapy for muscular diseases
TW202140787A (zh) 2020-02-28 2021-11-01 美商Ionis製藥公司 用於調節smn2之化合物及方法
KR20230009383A (ko) 2020-04-09 2023-01-17 아쏘씨아씨옹 엥스띠뛰 드 미올로지 근위축성 측삭경화증을 치료하기 위한 안티센스 서열
EP3978608A1 (en) 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
CA3233242A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
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US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
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JP2003528024A (ja) 1997-12-16 2003-09-24 バレンティス・インコーポレーテッド 製剤化された核酸分子の無針注入
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ES2694726T3 (es) * 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
JP2012509366A (ja) * 2008-11-17 2012-04-19 エンゾン ファーマシューティカルズ,インコーポレーテッド 核酸送達系のための放出可能ポリマー脂質
CN109576268A (zh) * 2009-04-10 2019-04-05 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
CN103477273A (zh) 2011-02-25 2013-12-25 卢米泰克斯公司 显示器前方照明装置

Also Published As

Publication number Publication date
WO2013053928A1 (en) 2013-04-18
EP2581448B1 (en) 2015-01-28
CA2776651A1 (en) 2013-04-13
DK2581448T3 (en) 2015-04-27
AU2012322903B2 (en) 2017-09-14
BR112014009066B8 (pt) 2021-02-23
EP2766479A1 (en) 2014-08-20
IL231983A0 (en) 2014-05-28
HK1186205A1 (en) 2014-03-07
CN104245935A (zh) 2014-12-24
ES2651216T3 (es) 2018-01-25
EP2766479B1 (en) 2017-09-13
IL231983B (en) 2018-04-30
EP2581448A1 (en) 2013-04-17
AU2012322903A1 (en) 2014-05-29
JP2015501144A (ja) 2015-01-15
BR112014009066B1 (pt) 2020-08-25
CA2776651C (en) 2021-06-08
US20140296323A1 (en) 2014-10-02
US9738891B2 (en) 2017-08-22
JP6181653B2 (ja) 2017-08-16
IN2014DN03463A (pl) 2015-06-05
BR112014009066A8 (pt) 2018-01-09
BR112014009066A2 (pt) 2017-04-18
CN104245935B (zh) 2018-05-08
PT2581448E (pt) 2015-05-21
CA2851970A1 (en) 2013-04-18
ES2535654T3 (es) 2015-05-13

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