DK3387130T3 - Effektiv genterapiværktøjer til skipning af exon 53 i dystrofingenet - Google Patents

Effektiv genterapiværktøjer til skipning af exon 53 i dystrofingenet Download PDF

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Publication number
DK3387130T3
DK3387130T3 DK16825816.8T DK16825816T DK3387130T3 DK 3387130 T3 DK3387130 T3 DK 3387130T3 DK 16825816 T DK16825816 T DK 16825816T DK 3387130 T3 DK3387130 T3 DK 3387130T3
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Denmark
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gene
exon
shipping
dystrophy
effective
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DK16825816.8T
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English (en)
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France Pietri-Rouxel
Virginie Francois
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Genethon
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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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • C12N15/62DNA sequences coding for fusion proteins
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
DK16825816.8T 2015-12-09 2016-12-09 Effektiv genterapiværktøjer til skipning af exon 53 i dystrofingenet DK3387130T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR1562036A FR3044926B1 (fr) 2015-12-09 2015-12-09 Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
PCT/FR2016/053312 WO2017098187A1 (fr) 2015-12-09 2016-12-09 Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine

Publications (1)

Publication Number Publication Date
DK3387130T3 true DK3387130T3 (da) 2020-12-07

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Family Applications (1)

Application Number Title Priority Date Filing Date
DK16825816.8T DK3387130T3 (da) 2015-12-09 2016-12-09 Effektiv genterapiværktøjer til skipning af exon 53 i dystrofingenet

Country Status (11)

Country Link
US (1) US10752898B2 (da)
EP (1) EP3387130B1 (da)
JP (1) JP6878433B2 (da)
CN (1) CN108368508B (da)
CA (1) CA3006515A1 (da)
DK (1) DK3387130T3 (da)
ES (1) ES2845214T3 (da)
FR (1) FR3044926B1 (da)
HK (1) HK1255100A1 (da)
PT (1) PT3387130T (da)
WO (1) WO2017098187A1 (da)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019146621A1 (ja) * 2018-01-25 2019-08-01 国立大学法人大阪大学 ペリオスチンの発現量増加またはスプライシングバリアントの変化を伴う疾患の治療用医薬組成物
EP3815696A4 (en) * 2018-06-26 2022-11-30 Nippon Shinyaku Co., Ltd. COMPOSITION COMPRISING AN ANTISENSE OLIGONUCLEOTIDE AND ITS USE FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108289A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
KR20210081322A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 디스트로핀병증을 치료하기 위한 그의 용도
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
CN113088531B (zh) * 2021-03-25 2023-10-17 四川省药品检验研究院(四川省医疗器械检测中心) 牛源性成分定量分析标准质粒、制备及检测方法及应用
CN113355409A (zh) * 2021-07-05 2021-09-07 北京华瑞康源生物科技发展有限公司 一种检测人dmd基因外显子拷贝数变异的引物及探针组合物和试剂盒
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023130959A1 (zh) * 2022-01-04 2023-07-13 广州瑞风生物科技有限公司 靶向USH2A pre-mRNA的snRNA及其应用

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
NZ600121A (en) 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
AU2002360291A1 (en) * 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
JP5054975B2 (ja) 2003-09-30 2012-10-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途
EP3228711A1 (en) 2004-06-28 2017-10-11 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
JP5963678B2 (ja) * 2009-11-12 2016-08-03 ジ ユニバーシティ オブ ウェスタン オーストラリア 病状を治療するためのアンチセンス分子及び方法
CA2792696C (en) 2010-03-17 2020-01-07 Association Institut De Myologie Modified u7 snrnas for treatment of neuromuscular diseases
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
BR112014033004B1 (pt) * 2012-07-03 2021-10-19 Biomarin Technologies B.V. Oligonucleotídeo para tratamento de pacientes com distrofia muscular
JP2016502858A (ja) * 2012-12-20 2016-02-01 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エキソンスキッピング組成物
US20160040162A1 (en) * 2013-03-14 2016-02-11 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US10131910B2 (en) * 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2

Also Published As

Publication number Publication date
WO2017098187A1 (fr) 2017-06-15
FR3044926A1 (fr) 2017-06-16
US10752898B2 (en) 2020-08-25
HK1255100A1 (zh) 2019-08-02
FR3044926B1 (fr) 2020-01-31
CA3006515A1 (fr) 2017-06-15
PT3387130T (pt) 2020-11-20
CN108368508B (zh) 2022-07-05
JP6878433B2 (ja) 2021-05-26
EP3387130A1 (fr) 2018-10-17
CN108368508A (zh) 2018-08-03
JP2019502376A (ja) 2019-01-31
US20180362980A1 (en) 2018-12-20
ES2845214T3 (es) 2021-07-26
EP3387130B1 (fr) 2020-11-11

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