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PT2344637E - Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne - Google Patents

Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne Download PDF

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Publication number
PT2344637E
PT2344637E PT09788160T PT09788160T PT2344637E PT 2344637 E PT2344637 E PT 2344637E PT 09788160 T PT09788160 T PT 09788160T PT 09788160 T PT09788160 T PT 09788160T PT 2344637 E PT2344637 E PT 2344637E
Authority
PT
Portugal
Prior art keywords
exon
mrna
methods
muscular dystrophy
duchenne muscular
Prior art date
Application number
PT09788160T
Other languages
English (en)
Inventor
Garrit-Jan Boudewijn Van Ommen
Gerardus Johannes Platenburg
Josephus Johannes De Kimpe
Judith Christina Theodora Van Deutekom
Annemieke Aartsma-Rus
Original Assignee
Academisch Ziekenhuis Leiden
Prosensa Technologies Bv
Prosensa Bv
Prosensa Holding Bv
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40873329&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PT2344637(E) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/NL2008/050673 external-priority patent/WO2009054725A2/en
Application filed by Academisch Ziekenhuis Leiden, Prosensa Technologies Bv, Prosensa Bv, Prosensa Holding Bv filed Critical Academisch Ziekenhuis Leiden
Publication of PT2344637E publication Critical patent/PT2344637E/pt

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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
    • 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
    • A61P21/04Drugs 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
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/3212'-O-R Modification

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
PT09788160T 2008-10-27 2009-01-13 Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne PT2344637E (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/NL2008/050673 WO2009054725A2 (en) 2007-10-26 2008-10-27 Means and methods for counteracting muscle disorders

Publications (1)

Publication Number Publication Date
PT2344637E true PT2344637E (pt) 2015-03-23

Family

ID=40873329

Family Applications (2)

Application Number Title Priority Date Filing Date
PT131603383T PT2607484E (pt) 2008-10-27 2009-01-13 Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
PT09788160T PT2344637E (pt) 2008-10-27 2009-01-13 Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT131603383T PT2607484E (pt) 2008-10-27 2009-01-13 Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne

Country Status (15)

Country Link
EP (4) EP2607484B1 (pt)
JP (2) JP5786109B2 (pt)
AU (1) AU2009310557B2 (pt)
CA (3) CA2741629C (pt)
CY (1) CY1116305T1 (pt)
DK (2) DK2344637T4 (pt)
ES (2) ES2532634T5 (pt)
HK (1) HK1160169A1 (pt)
HR (1) HRP20160078T1 (pt)
IL (2) IL212509A (pt)
NZ (1) NZ592498A (pt)
PL (1) PL2607484T3 (pt)
PT (2) PT2607484E (pt)
SI (1) SI2607484T1 (pt)
WO (2) WO2010050801A1 (pt)

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EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP3428278A1 (en) 2008-10-24 2019-01-16 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
WO2011036640A2 (en) * 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
PT2499249T (pt) 2009-11-12 2018-11-21 Univ Western Australia Moléculas antisentido e métodos para tratar patologias
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WO2012109296A1 (en) * 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
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CN112251436A (zh) * 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
EP3608407A1 (en) * 2012-07-03 2020-02-12 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
CN105378081B (zh) 2013-03-14 2019-06-14 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
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
US11236338B2 (en) * 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
JP6519842B2 (ja) * 2013-10-04 2019-05-29 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸
UA120849C2 (uk) * 2014-03-12 2020-02-25 Ніппон Шин'Яку Ко., Лтд. Антисенсовий олігомер
HRP20220379T1 (hr) 2014-06-10 2022-05-27 Erasmus University Medical Center Rotterdam Antisense oligonukleotidi korisni u liječenju pompeove bolesti
PL3351633T3 (pl) * 2015-09-15 2020-11-02 Nippon Shinyaku Co., Ltd. Antysensowny kwas nukleinowy
US20190127733A1 (en) 2015-10-09 2019-05-02 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
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US11339392B2 (en) 2016-03-02 2022-05-24 The Board Of Trustees Of The Leland Stanford Junior University Pan-genotypic agents against influenza virus and methods of using the same
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MA45290A (fr) * 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
MX2018016052A (es) 2016-06-30 2019-05-02 Sarepta Therapeutics Inc Oligomeros de omision de exon para distrofia muscular.
AU2017291960B2 (en) * 2016-07-05 2024-03-14 Biomarin Technologies B.V. Pre-mRNA splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
MX2019006882A (es) 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
MX2019006989A (es) 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
EP4115908A1 (en) 2016-12-19 2023-01-11 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR20190104381A (ko) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
WO2019071028A1 (en) 2017-10-04 2019-04-11 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
EP3720448A4 (en) 2017-12-06 2021-11-03 Avidity Biosciences, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP2021526796A (ja) * 2018-06-13 2021-10-11 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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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KR20210081322A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도
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Also Published As

Publication number Publication date
IL212509A0 (en) 2011-06-30
EP3400948B1 (en) 2022-11-02
IL212508A0 (en) 2011-06-30
JP5786109B2 (ja) 2015-09-30
CA2741793A1 (en) 2010-05-06
WO2010050802A2 (en) 2010-05-06
CA3017539A1 (en) 2010-05-06
AU2009310558A1 (en) 2010-05-06
WO2010050802A3 (en) 2010-08-12
CA2741793C (en) 2018-10-30
JP2012506697A (ja) 2012-03-22
EP2344637B1 (en) 2014-12-24
EP2344637B2 (en) 2018-02-28
CA2741629C (en) 2022-07-05
DK2344637T3 (en) 2015-02-02
EP2607484B1 (en) 2016-01-06
ES2532634T5 (es) 2018-04-30
AU2009310557B2 (en) 2014-09-11
DK2344637T4 (en) 2018-04-23
CA2741629A1 (en) 2010-05-06
HK1160169A1 (en) 2012-08-10
ES2532634T3 (es) 2015-03-30
AU2009310558B2 (en) 2014-09-04
EP2344637A1 (en) 2011-07-20
IL212509A (en) 2016-04-21
CY1116305T1 (el) 2017-02-08
HRP20160078T1 (hr) 2016-04-08
JP5905260B2 (ja) 2016-04-20
EP2349287B1 (en) 2018-08-15
IL212508A (en) 2013-11-28
EP3400948A1 (en) 2018-11-14
JP2012506698A (ja) 2012-03-22
NZ592498A (en) 2012-05-25
PT2607484E (pt) 2016-03-09
EP2607484A1 (en) 2013-06-26
DK2607484T3 (en) 2016-03-07
AU2009310558A8 (en) 2015-01-22
EP2349287A2 (en) 2011-08-03
SI2607484T1 (sl) 2016-11-30
AU2009310557A1 (en) 2010-05-06
PL2607484T3 (pl) 2016-06-30
AU2009310557A8 (en) 2011-09-29
ES2562658T3 (es) 2016-03-07
WO2010050801A1 (en) 2010-05-06

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