HK1249106A1 - 反义诱导之酸性α-葡萄苷酶中的外显子2包含 - Google Patents

反义诱导之酸性α-葡萄苷酶中的外显子2包含 Download PDF

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Publication number
HK1249106A1
HK1249106A1 HK18108560.3A HK18108560A HK1249106A1 HK 1249106 A1 HK1249106 A1 HK 1249106A1 HK 18108560 A HK18108560 A HK 18108560A HK 1249106 A1 HK1249106 A1 HK 1249106A1
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HK
Hong Kong
Prior art keywords
glucosidase
inclusion
acid alpha
antisense
deficiency
Prior art date
Application number
HK18108560.3A
Other languages
English (en)
Chinese (zh)
Inventor
Stephen Donald Wilton
Sue Fletcher
Gunnar James Hanson
Richard Keith Bestwick
Frederick J. Schnell
Original Assignee
Sarepta Therapeutics, Inc.
Murdoch University
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 Sarepta Therapeutics, Inc., Murdoch University filed Critical Sarepta Therapeutics, Inc.
Publication of HK1249106A1 publication Critical patent/HK1249106A1/zh

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Emergency Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
HK18108560.3A 2015-02-27 2016-02-29 反义诱导之酸性α-葡萄苷酶中的外显子2包含 HK1249106A1 (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562126346P 2015-02-27 2015-02-27
US62/126,346 2015-02-27
US201562234263P 2015-09-29 2015-09-29
US62/234,263 2015-09-29
US201662300635P 2016-02-26 2016-02-26
US62/300,635 2016-02-26
PCT/US2016/020127 WO2016138534A2 (en) 2015-02-27 2016-02-29 Antisense-induced exon2 inclusion in acid alpha-glucosidase

Publications (1)

Publication Number Publication Date
HK1249106A1 true HK1249106A1 (zh) 2018-10-26

Family

ID=56789314

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18108560.3A HK1249106A1 (zh) 2015-02-27 2016-02-29 反义诱导之酸性α-葡萄苷酶中的外显子2包含

Country Status (12)

Country Link
US (2) US20180216111A1 (enExample)
EP (1) EP3262056A4 (enExample)
JP (4) JP2018509143A (enExample)
AU (2) AU2016224976A1 (enExample)
BR (1) BR112017018383B1 (enExample)
CA (1) CA2977528A1 (enExample)
HK (1) HK1249106A1 (enExample)
IL (2) IL254112B (enExample)
MA (1) MA41759A (enExample)
MX (2) MX2017011004A (enExample)
TW (2) TW202403045A (enExample)
WO (1) WO2016138534A2 (enExample)

Families Citing this family (28)

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US20150197534A1 (en) 2013-09-05 2015-07-16 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
CA2986228A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11083745B2 (en) 2015-12-14 2021-08-10 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP2018538302A (ja) * 2015-12-15 2018-12-27 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチド複合体
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) * 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
SMT202400071T1 (it) 2017-08-25 2024-03-13 Stoke Therapeutics Inc Oligomeri antisenso per il trattamento di condizioni e malattie
EP3784248A4 (en) * 2018-04-26 2022-08-10 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
CA3173647A1 (en) 2020-05-11 2021-11-18 Isabel AZNAREZ Opa1 antisense oligomers for treatment of conditions and diseases
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023283629A1 (en) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations thereof for treating facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024537711A (ja) * 2021-09-30 2024-10-16 サレプタ セラピューティクス， インコーポレイテッド １つ以上の脱塩基ユニットを有するアンチセンスオリゴヌクレオチド
AU2023254846A1 (en) 2022-04-15 2024-10-10 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
JP2025524656A (ja) 2022-07-14 2025-07-30 ザ・ブロード・インスティテュート・インコーポレイテッド トランスフェリン受容体との相互作用を通じてｃｎｓ全体への遺伝子送達を可能にするａａｖキャプシド
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4914224B2 (ja) * 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
NZ603606A (en) * 2010-05-28 2015-06-26 Sarepta Therapeutics Inc Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP6478632B2 (ja) * 2011-05-05 2019-03-06 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2012340390B2 (en) * 2011-11-18 2016-11-24 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
NZ631245A (en) * 2013-03-14 2017-09-29 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
US20150197534A1 (en) * 2013-09-05 2015-07-16 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
CA2948373A1 (en) * 2014-05-16 2015-11-19 Oregon State University Antisense antibacterial compounds and methods
US10308940B2 (en) * 2014-06-10 2019-06-04 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of Pompe disease

Also Published As

Publication number Publication date
JP2018509143A (ja) 2018-04-05
EP3262056A2 (en) 2018-01-03
JP2023129494A (ja) 2023-09-14
EP3262056A4 (en) 2018-09-19
IL281199B (en) 2022-05-01
IL254112B (en) 2021-04-29
MX2017011004A (es) 2018-02-09
MA41759A (fr) 2018-01-03
BR112017018383A2 (pt) 2018-09-04
IL254112A (en) 2018-06-28
WO2016138534A3 (en) 2016-12-22
TW202403045A (zh) 2024-01-16
BR112017018383B1 (pt) 2023-04-25
AU2020203825A1 (en) 2020-07-02
JP2024074908A (ja) 2024-05-31
CA2977528A1 (en) 2016-09-01
IL281199A (en) 2021-04-29
AU2020203825B2 (en) 2021-08-05
AU2016224976A1 (en) 2017-09-14
JP2021166543A (ja) 2021-10-21
US20250171783A1 (en) 2025-05-29
WO2016138534A2 (en) 2016-09-01
TW201702378A (zh) 2017-01-16
MX2024010190A (es) 2024-08-28
US20180216111A1 (en) 2018-08-02

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