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MX2017011004A - Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. - Google Patents

Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.

Info

Publication number
MX2017011004A
MX2017011004A MX2017011004A MX2017011004A MX2017011004A MX 2017011004 A MX2017011004 A MX 2017011004A MX 2017011004 A MX2017011004 A MX 2017011004A MX 2017011004 A MX2017011004 A MX 2017011004A MX 2017011004 A MX2017011004 A MX 2017011004A
Authority
MX
Mexico
Prior art keywords
glucosidase
inclusion
acid alpha
antisense
deficiency
Prior art date
Application number
MX2017011004A
Other languages
English (en)
Inventor
Donald Wilton Stephen
Fletcher Sue
James Hanson Gunnar
Keith Bestwick Richard
J Schnell Frederick
Original Assignee
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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of MX2017011004A publication Critical patent/MX2017011004A/es

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Classifications

    • 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)
  • 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)

Abstract

La presente divulgación se refiere a oligómeros de antisentido y composiciones y métodos relacionados para inducir la inclusión de un exón como un tratamiento para la enfermedad de almacenamiento de glucógeno tipo II (GSD II) (conocida también como enfermedad de Pompe, glucogenosis II, deficiencia de maltasa ácida (AMD), deficiencia de alfa-glucosidasa ácida y deficiencia de alfa-glucosidasa lisosomal), y más específicamente se refiere a la inducción de la inclusión del exón 2 y con ello restaurar los niveles de la proteína de alfa-glucosidasa ácida (GAA) enzimáticamente activa codificada por el gen GAA.
MX2017011004A 2015-02-27 2016-02-29 Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. MX2017011004A (es)

Applications Claiming Priority (4)

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
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
MX2017011004A true MX2017011004A (es) 2018-02-09

Family

ID=56789314

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017011004A MX2017011004A (es) 2015-02-27 2016-02-29 Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.
MX2024010190A MX2024010190A (es) 2015-02-27 2017-08-25 Inclusion del exon2 inducida por antisentido en alfa-glucosidasa acida.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024010190A MX2024010190A (es) 2015-02-27 2017-08-25 Inclusion del exon2 inducida por antisentido en alfa-glucosidasa acida.

Country Status (12)

Country Link
US (2) US20180216111A1 (es)
EP (1) EP3262056A4 (es)
JP (4) JP2018509143A (es)
AU (2) AU2016224976A1 (es)
BR (1) BR112017018383B1 (es)
CA (1) CA2977528A1 (es)
HK (1) HK1249106A1 (es)
IL (2) IL254112B (es)
MA (1) MA41759A (es)
MX (2) MX2017011004A (es)
TW (2) TW201702378A (es)
WO (1) WO2016138534A2 (es)

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CN111235149B (zh) 2013-09-05 2024-04-16 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US9976143B2 (en) 2014-10-03 2018-05-22 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3297649B1 (en) 2015-05-19 2023-10-11 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
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
MX2018007307A (es) * 2015-12-15 2019-03-14 Sarepta Therapeutics Inc Conjugados de peptidos y oligonucleotidos.
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
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
GB2594767B (en) 2017-08-25 2022-06-22 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2020028864A1 (en) 2018-08-02 2020-02-06 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
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
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
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
IL311568A (en) * 2021-09-30 2024-05-01 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
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
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011150408A2 (en) * 2010-05-28 2011-12-01 Avi Biopharma, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
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
CN108864192A (zh) * 2011-11-18 2018-11-23 萨勒普塔医疗公司 功能改性的寡核苷酸及其亚单元
CN105378081B (zh) * 2013-03-14 2019-06-14 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
CN111235149B (zh) * 2013-09-05 2024-04-16 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
EP3143141B1 (en) * 2014-05-16 2019-10-02 Oregon State University Antisense antibacterial compounds and methods
CN106661580B (zh) * 2014-06-10 2022-02-15 鹿特丹伊拉斯谟大学医疗中心 用于治疗庞帕病的反义寡核苷酸

Also Published As

Publication number Publication date
TW201702378A (zh) 2017-01-16
AU2016224976A1 (en) 2017-09-14
EP3262056A4 (en) 2018-09-19
JP2023129494A (ja) 2023-09-14
TW202403045A (zh) 2024-01-16
JP2021166543A (ja) 2021-10-21
WO2016138534A2 (en) 2016-09-01
JP2024074908A (ja) 2024-05-31
WO2016138534A3 (en) 2016-12-22
IL254112B (en) 2021-04-29
MX2024010190A (es) 2024-08-28
BR112017018383A2 (pt) 2018-09-04
US20250171783A1 (en) 2025-05-29
HK1249106A1 (zh) 2018-10-26
IL254112A (en) 2018-06-28
JP2018509143A (ja) 2018-04-05
AU2020203825B2 (en) 2021-08-05
IL281199B (en) 2022-05-01
CA2977528A1 (en) 2016-09-01
AU2020203825A1 (en) 2020-07-02
IL281199A (en) 2021-04-29
US20180216111A1 (en) 2018-08-02
MA41759A (fr) 2018-01-03
BR112017018383B1 (pt) 2023-04-25
EP3262056A2 (en) 2018-01-03

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