JP2018509143A - 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ - Google Patents

酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ Download PDF

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JP2018509143A
JP2018509143A JP2017545273A JP2017545273A JP2018509143A JP 2018509143 A JP2018509143 A JP 2018509143A JP 2017545273 A JP2017545273 A JP 2017545273A JP 2017545273 A JP2017545273 A JP 2017545273A JP 2018509143 A JP2018509143 A JP 2018509143A
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seq
alkyl
formula
iii
cpp
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JP2018509143A5 (https=
Inventor
スティーブン ドナルド ウィルトン，
スティーブン ドナルド ウィルトン，
スー フレッチャー，
スー フレッチャー，
ガンナー ジェイムズ ハンソン，
ガンナー ジェイムズ ハンソン，
リチャード キース ベストウィック，
リチャード キース ベストウィック，
フレデリック ジェイ． シュネル，
フレデリック ジェイ． シュネル，
Original Assignee
サレプタ セラピューティクス，インコーポレイテッド
サレプタ セラピューティクス，インコーポレイテッド
マードック ユニバーシティ
マードック ユニバーシティ
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Publication of JP2018509143A publication Critical patent/JP2018509143A/ja
Publication of JP2018509143A5 publication Critical patent/JP2018509143A5/ja
Priority to JP2021114792A priority Critical patent/JP2021166543A/ja
Priority to JP2023115841A priority patent/JP2023129494A/ja
Priority to JP2024060083A priority patent/JP2024074908A/ja
Ceased legal-status Critical Current

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    • 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
    • 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
    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2017545273A 2015-02-27 2016-02-29 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ Ceased JP2018509143A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2021114792A JP2021166543A (ja) 2015-02-27 2021-07-12 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2023115841A JP2023129494A (ja) 2015-02-27 2023-07-14 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2024060083A JP2024074908A (ja) 2015-02-27 2024-04-03 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021114792A Division JP2021166543A (ja) 2015-02-27 2021-07-12 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ

Publications (2)

Publication Number Publication Date
JP2018509143A true JP2018509143A (ja) 2018-04-05
JP2018509143A5 JP2018509143A5 (https=) 2019-04-11

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JP2017545273A Ceased JP2018509143A (ja) 2015-02-27 2016-02-29 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2021114792A Pending JP2021166543A (ja) 2015-02-27 2021-07-12 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2023115841A Pending JP2023129494A (ja) 2015-02-27 2023-07-14 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2024060083A Pending JP2024074908A (ja) 2015-02-27 2024-04-03 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2021114792A Pending JP2021166543A (ja) 2015-02-27 2021-07-12 酸性アルファ−グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2023115841A Pending JP2023129494A (ja) 2015-02-27 2023-07-14 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ
JP2024060083A Pending JP2024074908A (ja) 2015-02-27 2024-04-03 酸性アルファ－グルコシダーゼにおけるアンチセンスに誘導されるエクソン２の組入れ

Country Status (12)

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

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GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016264471B2 (en) 2015-05-19 2022-01-27 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
BR112018012237A2 (pt) 2015-12-15 2018-12-04 Sarepta Therapeutics Inc conjugados oligonucleotídeos-peptídeos
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
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
JP2021521794A (ja) * 2018-04-26 2021-08-30 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およびオリゴマーコンジュゲート
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
TW202449155A (zh) * 2018-07-27 2024-12-16 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
SG11202100928QA (en) 2018-08-02 2021-02-25 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
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
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
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023055774A1 (en) * 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
AU2023307223A1 (en) 2022-07-14 2025-02-20 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
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
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
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

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WO2015035231A1 (en) * 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2015190922A1 (en) * 2014-06-10 2015-12-17 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease

Also Published As

Publication number Publication date
TW202403045A (zh) 2024-01-16
US20250171783A1 (en) 2025-05-29
WO2016138534A3 (en) 2016-12-22
MA41759A (fr) 2018-01-03
TW201702378A (zh) 2017-01-16
JP2023129494A (ja) 2023-09-14
JP2021166543A (ja) 2021-10-21
EP3262056A2 (en) 2018-01-03
US20180216111A1 (en) 2018-08-02
IL254112A (en) 2018-06-28
BR112017018383B1 (pt) 2023-04-25
EP3262056A4 (en) 2018-09-19
WO2016138534A2 (en) 2016-09-01
IL281199A (en) 2021-04-29
CA2977528A1 (en) 2016-09-01
MX2024010190A (es) 2024-08-28
HK1249106A1 (zh) 2018-10-26
IL281199B (en) 2022-05-01
MX2017011004A (es) 2018-02-09
JP2024074908A (ja) 2024-05-31
AU2020203825A1 (en) 2020-07-02
IL254112B (en) 2021-04-29
BR112017018383A2 (pt) 2018-09-04
AU2016224976A1 (en) 2017-09-14
AU2020203825B2 (en) 2021-08-05

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