MA41759A - Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide - Google Patents

Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide

Info

Publication number
MA41759A
MA41759A MA041759A MA41759A MA41759A MA 41759 A MA41759 A MA 41759A MA 041759 A MA041759 A MA 041759A MA 41759 A MA41759 A MA 41759A MA 41759 A MA41759 A MA 41759A
Authority
MA
Morocco
Prior art keywords
glucosidase
inclusion
exon
antisens
induced
Prior art date
Application number
MA041759A
Other languages
English (en)
Inventor
Richard Keith Bestwick
Sue Fletcher
Gunnar James Hanson
Frederick J Schnell
Stephen Donald Wilton
Original Assignee
Univ Murdoch
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 Univ Murdoch, Sarepta Therapeutics Inc filed Critical Univ Murdoch
Publication of MA41759A publication Critical patent/MA41759A/fr

Links

Classifications

    • 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

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)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Obesity (AREA)
  • Veterinary Medicine (AREA)
  • Endocrinology (AREA)
  • Emergency 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)

Abstract

La présente invention concerne des oligomères antisens et des compositions apparentées et des procédés d'induction d'inclusion d'exon en tant que traitement pour la maladie de stockage du glycogène de type ii (gsd-ii) (également appelée maladie de pompe, glycogénose ii, déficit en acide maltase, déficience en glucosidase, et déficience en alpha-glucosidase lysosomale), et plus spécifiquement concerne l'induction de l'inclusion de l'exon 2 et ainsi la restauration des niveaux de la protéine alpha-glucosidase acide (gaa) enzymatiquement active codée par le gène gaa.
MA041759A 2015-02-27 2016-02-29 Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide MA41759A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562126346P 2015-02-27 2015-02-27
US201562234263P 2015-09-29 2015-09-29
US201662300635P 2016-02-26 2016-02-26

Publications (1)

Publication Number Publication Date
MA41759A true MA41759A (fr) 2018-01-03

Family

ID=56789314

Family Applications (1)

Application Number Title Priority Date Filing Date
MA041759A MA41759A (fr) 2015-02-27 2016-02-29 Inclusion de l'exon 2, induite par antisens, dans une alpha-glucosidase acide

Country Status (12)

Country Link
US (1) US20180216111A1 (fr)
EP (1) EP3262056A4 (fr)
JP (3) JP2018509143A (fr)
AU (2) AU2016224976A1 (fr)
BR (1) BR112017018383B1 (fr)
CA (1) CA2977528A1 (fr)
HK (1) HK1249106A1 (fr)
IL (2) IL254112B (fr)
MA (1) MA41759A (fr)
MX (1) MX2017011004A (fr)
TW (2) TW202403045A (fr)
WO (1) WO2016138534A2 (fr)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR102620328B1 (ko) 2014-10-03 2024-01-02 콜드스프링하버러보러토리 핵 유전자 산출량의 표적화 증강
US10675356B2 (en) 2015-05-19 2020-06-09 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
EP3390636B1 (fr) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Oligomères antisens destinés au traitement du syndrome de dravet
CA3006748A1 (fr) 2015-12-15 2017-06-22 Sarepta Therapeutics, Inc. Conjugues peptide-oligonucleotide
JP7033547B2 (ja) 2016-04-18 2022-03-10 サレプタ セラピューティクス， インコーポレイテッド 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
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.
PT3673080T (pt) 2017-08-25 2023-12-06 Stoke Therapeutics Inc Oligómeros anti-sentido para o tratamento de estados patológicos e outras doenças
EP3784248A4 (fr) * 2018-04-26 2022-08-10 Sarepta Therapeutics, Inc. Oligomères induisant le saut d'exon et conjugués d'oligomères pour la dystrophie musculaire
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
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 (fr) * 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Complexes de ciblage musculaire et leurs formulations pour traiter la dystrophie musculaire facio-scapulo-humérale
WO2023055774A1 (fr) * 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Oligonucléotides antisens ayant une ou plusieurs unités abasiques
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
WO2024016003A2 (fr) 2022-07-14 2024-01-18 The Broad Institute, Inc. Capsides d'aav qui permettent une distribution de gènes à l'échelle du snc par l'intermédiaire d'interactions avec le récepteur de transferrine

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
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US9161948B2 (en) * 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102339196B1 (ko) * 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9278987B2 (en) * 2011-11-18 2016-03-08 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
BR112015023001B8 (pt) * 2013-03-14 2022-08-09 Sarepta Therapeutics Inc Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
AU2014317961B2 (en) * 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
AU2015258895A1 (en) * 2014-05-16 2016-11-24 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
KR102524543B1 (ko) * 2014-06-10 2023-04-20 에라스무스 유니버시티 메디컬 센터 로테르담 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드

Also Published As

Publication number Publication date
BR112017018383B1 (pt) 2023-04-25
BR112017018383A2 (pt) 2018-09-04
TW202403045A (zh) 2024-01-16
MX2017011004A (es) 2018-02-09
AU2020203825B2 (en) 2021-08-05
US20180216111A1 (en) 2018-08-02
AU2016224976A1 (en) 2017-09-14
IL281199B (en) 2022-05-01
CA2977528A1 (fr) 2016-09-01
HK1249106A1 (zh) 2018-10-26
JP2021166543A (ja) 2021-10-21
IL281199A (en) 2021-04-29
IL254112A (en) 2018-06-28
JP2023129494A (ja) 2023-09-14
AU2020203825A1 (en) 2020-07-02
TW201702378A (zh) 2017-01-16
EP3262056A4 (fr) 2018-09-19
EP3262056A2 (fr) 2018-01-03
WO2016138534A3 (fr) 2016-12-22
IL254112B (en) 2021-04-29
JP2018509143A (ja) 2018-04-05
WO2016138534A2 (fr) 2016-09-01

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