CA2977528A1 - Antisense-induced exon2 inclusion in acid alpha-glucosidase - Google Patents

Antisense-induced exon2 inclusion in acid alpha-glucosidase Download PDF

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Publication number
CA2977528A1
CA2977528A1 CA2977528A CA2977528A CA2977528A1 CA 2977528 A1 CA2977528 A1 CA 2977528A1 CA 2977528 A CA2977528 A CA 2977528A CA 2977528 A CA2977528 A CA 2977528A CA 2977528 A1 CA2977528 A1 CA 2977528A1
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CA
Canada
Prior art keywords
seq
cxg
ggg
ccc
gcx
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Legal status (The legal status 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 status listed.)
Pending
Application number
CA2977528A
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English (en)
French (fr)
Inventor
Stephen Donald Wilton
Sue Fletcher
Gunnar James Hanson
Richard Keith Bestwick
Frederick J. Schnell
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Murdoch University
Sarepta Therapeutics Inc
Original Assignee
Murdoch University
Sarepta Therapeutics Inc
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 Murdoch University, Sarepta Therapeutics Inc filed Critical Murdoch University
Publication of CA2977528A1 publication Critical patent/CA2977528A1/en
Pending 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Endocrinology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Emergency Medicine (AREA)
  • General Chemical & Material Sciences (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
CA2977528A 2015-02-27 2016-02-29 Antisense-induced exon2 inclusion in acid alpha-glucosidase Pending CA2977528A1 (en)

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
CA2977528A1 true CA2977528A1 (en) 2016-09-01

Family

ID=56789314

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2977528A Pending CA2977528A1 (en) 2015-02-27 2016-02-29 Antisense-induced exon2 inclusion in acid alpha-glucosidase

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 (3) TW202403045A (enExample)
WO (1) WO2016138534A2 (enExample)

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AU2014317961B2 (en) 2013-09-05 2020-07-30 Murdoch University 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
KR102644053B1 (ko) 2015-05-19 2024-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
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
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EA201891419A1 (ru) 2015-12-15 2019-01-31 Сарепта Терапьютикс, Инк. Пептид-олигонуклеотидные конъюгаты
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
NL2017295B1 (en) * 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
KR20260009385A (ko) 2017-08-25 2026-01-19 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
WO2019209764A2 (en) * 2018-04-26 2019-10-31 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
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
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF 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
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses 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
WO2023283629A1 (en) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations thereof for treating facioscapulohumeral muscular dystrophy
US20240425864A1 (en) * 2021-09-30 2024-12-26 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
EP4554961A2 (en) 2022-07-14 2025-05-21 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
WO2026107393A2 (en) 2024-11-14 2026-05-21 The Broad Institute, Inc. Aav capsid modifications that reduce immunogenicity

Family Cites Families (10)

* Cited by examiner, † Cited by third party
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DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
KR101981705B1 (ko) * 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2011367230B2 (en) * 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR20140097398A (ko) * 2011-11-18 2014-08-06 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
US9217148B2 (en) * 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
AU2014317961B2 (en) * 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
US9790495B2 (en) * 2014-05-16 2017-10-17 Oregon State University Antisense antibacterial compounds and methods
EP4039807A1 (en) * 2014-06-10 2022-08-10 Erasmus University Rotterdam Medical Center Antisense oligonucleotides useful in treatment of pompe disease

Also Published As

Publication number Publication date
TW202403045A (zh) 2024-01-16
WO2016138534A3 (en) 2016-12-22
JP2024074908A (ja) 2024-05-31
IL281199B (en) 2022-05-01
IL254112A (en) 2018-06-28
WO2016138534A2 (en) 2016-09-01
BR112017018383A2 (pt) 2018-09-04
EP3262056A4 (en) 2018-09-19
TW201702378A (zh) 2017-01-16
MX2017011004A (es) 2018-02-09
MX2024010190A (es) 2024-08-28
JP2021166543A (ja) 2021-10-21
JP2018509143A (ja) 2018-04-05
TW202617995A (zh) 2026-05-01
HK1249106A1 (zh) 2018-10-26
AU2020203825A1 (en) 2020-07-02
IL281199A (en) 2021-04-29
MA41759A (fr) 2018-01-03
JP2023129494A (ja) 2023-09-14
IL254112B (en) 2021-04-29
AU2016224976A1 (en) 2017-09-14
BR112017018383B1 (pt) 2023-04-25
US20180216111A1 (en) 2018-08-02
AU2020203825B2 (en) 2021-08-05
EP3262056A2 (en) 2018-01-03
US20250171783A1 (en) 2025-05-29

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