HK1216902A1 - 用作治疗肌肉萎缩的改进外显子跳跃组合物 - Google Patents

用作治疗肌肉萎缩的改进外显子跳跃组合物 Download PDF

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Publication number
HK1216902A1
HK1216902A1 HK16104638.2A HK16104638A HK1216902A1 HK 1216902 A1 HK1216902 A1 HK 1216902A1 HK 16104638 A HK16104638 A HK 16104638A HK 1216902 A1 HK1216902 A1 HK 1216902A1
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Hong Kong
Prior art keywords
muscular dystrophy
exon skipping
treating muscular
skipping compositions
improved exon
Prior art date
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HK16104638.2A
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English (en)
Chinese (zh)
Inventor
Richard K. Bestwick
Diane Elizabeth FRANK
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Sarepta Therapeutics, Inc.
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Application filed by Sarepta Therapeutics, Inc. filed Critical Sarepta Therapeutics, Inc.
Publication of HK1216902A1 publication Critical patent/HK1216902A1/zh

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Plant Substances (AREA)
HK16104638.2A 2012-12-20 2013-12-20 用作治疗肌肉萎缩的改进外显子跳跃组合物 HK1216902A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261739968P 2012-12-20 2012-12-20
US201261739968P 2012-12-20
PCT/US2013/077216 WO2014100714A1 (en) 2012-12-20 2013-12-20 Improved exon skipping compositions for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
HK1216902A1 true HK1216902A1 (zh) 2016-12-09

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HK16104638.2A HK1216902A1 (zh) 2012-12-20 2013-12-20 用作治疗肌肉萎缩的改进外显子跳跃组合物

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Country Link
US (5) US20150361428A1 (enExample)
EP (2) EP3885439A1 (enExample)
JP (3) JP2016502858A (enExample)
KR (2) KR20200143739A (enExample)
CN (2) CN106414739A (enExample)
AU (3) AU2013364158A1 (enExample)
BR (1) BR112015014987A2 (enExample)
CA (1) CA2894899A1 (enExample)
EA (1) EA201591178A1 (enExample)
HK (1) HK1216902A1 (enExample)
IL (1) IL239491A0 (enExample)
MX (2) MX2015008035A (enExample)
WO (1) WO2014100714A1 (enExample)

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WO2014100714A1 (en) 2014-06-26
US20230104708A1 (en) 2023-04-06
EA201591178A1 (ru) 2015-11-30
JP2016502858A (ja) 2016-02-01
EP3885439A1 (en) 2021-09-29
JP2019050834A (ja) 2019-04-04
EP2935584A1 (en) 2015-10-28
KR20200143739A (ko) 2020-12-24
US20150361428A1 (en) 2015-12-17
CA2894899A1 (en) 2014-06-26
MX2015008035A (es) 2016-03-17
CN106414739A (zh) 2017-02-15
US20170369875A1 (en) 2017-12-28
US20220259592A1 (en) 2022-08-18
AU2013364158A1 (en) 2015-07-09
JP2018110601A (ja) 2018-07-19
AU2019253778A1 (en) 2019-11-07
AU2022201628A1 (en) 2022-03-31
CN111440796A (zh) 2020-07-24
MX2019008164A (es) 2019-09-13
KR20150099804A (ko) 2015-09-01
IL239491A0 (en) 2015-08-31
BR112015014987A2 (pt) 2017-08-15
US20200339985A1 (en) 2020-10-29

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