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 acideInfo
- 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
Links
- 108010028144 alpha-Glucosidases Proteins 0.000 title abstract 4
- 239000002253 acid Substances 0.000 title abstract 2
- 102100024295 Maltase-glucoamylase Human genes 0.000 title 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 abstract 3
- 102000016679 alpha-Glucosidases Human genes 0.000 abstract 3
- 230000007812 deficiency Effects 0.000 abstract 2
- 101150115151 GAA gene Proteins 0.000 abstract 1
- 102000004366 Glucosidases Human genes 0.000 abstract 1
- 108010056771 Glucosidases Proteins 0.000 abstract 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 abstract 1
- 230000002378 acidificating effect Effects 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000007345 glycogen storage disease Diseases 0.000 abstract 1
- 201000004502 glycogen storage disease II Diseases 0.000 abstract 1
- 230000006698 induction Effects 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
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)
- 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 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.
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 (2) | US20180216111A1 (fr) |
| EP (1) | EP3262056A4 (fr) |
| JP (4) | 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 (2) | MX2017011004A (fr) |
| TW (2) | TW202403045A (fr) |
| WO (1) | WO2016138534A2 (fr) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11236338B2 (en) | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| 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 (fr) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Oligomères antisens destinés au traitement du retard mental dominant autosomique 5 et du syndrome de dravet |
| BR112018012237A2 (pt) | 2015-12-15 | 2018-12-04 | Sarepta Therapeutics Inc | conjugados oligonucleotídeos-peptídeos |
| WO2017184529A1 (fr) | 2016-04-18 | 2017-10-26 | Sarepta Therapeutics, Inc. | Oligomères antisens et procédés d'utilisation de ceux-ci pour le traitement de maladies associées au gène de l'alpha-glucosidase acide |
| 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 (fr) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methodes et compositions pour le traitement d'une maladie de stockage d'ester de cholesteryle |
| 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 (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 |
| 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 (fr) * | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Oligonucléotides antisens ayant une ou plusieurs unités abasiques |
| 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 (fr) | 2024-01-17 | 2025-07-24 | The Broad Institute, Inc. | Modifications de capsides de vaa permettant une administration améliorée de gènes à l'échelle du snc par l'intermédiaire d'interactions avec le récepteur de la transferrine |
| WO2025217163A2 (fr) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Nouveaux capsides aav se liant à cd59 humain |
| WO2025217174A1 (fr) | 2024-04-08 | 2025-10-16 | The Broad Institute, Inc. | Modifications de capside d'aav qui permettent une délivrance améliorée de gènes à l'échelle du snc par l'intermédiaire d'interactions avec l'anhydrase carbonique iv |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2553955C (fr) * | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Alpha-glucosidase acide et fragments de celle-ci |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| KR20200133284A (ko) * | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| CA3092114A1 (fr) * | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Conjugues peptides/oligonucleotides |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| HK1201514A1 (en) * | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| CN110218727A (zh) * | 2013-03-14 | 2019-09-10 | 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 |
| US11236338B2 (en) * | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| CA2948373A1 (fr) * | 2014-05-16 | 2015-11-19 | Oregon State University | Composes antisens antibacteriens et procedes associes |
| KR102524543B1 (ko) * | 2014-06-10 | 2023-04-20 | 에라스무스 유니버시티 메디컬 센터 로테르담 | 폼페병의 치료에 유용한 안티센스 올리고뉴클레오티드 |
-
2016
- 2016-02-29 EP EP16756555.5A patent/EP3262056A4/fr active Pending
- 2016-02-29 US US15/553,911 patent/US20180216111A1/en not_active Abandoned
- 2016-02-29 HK HK18108560.3A patent/HK1249106A1/zh unknown
- 2016-02-29 BR BR112017018383-8A patent/BR112017018383B1/pt active IP Right Grant
- 2016-02-29 JP JP2017545273A patent/JP2018509143A/ja not_active Ceased
- 2016-02-29 MA MA041759A patent/MA41759A/fr unknown
- 2016-02-29 MX MX2017011004A patent/MX2017011004A/es unknown
- 2016-02-29 CA CA2977528A patent/CA2977528A1/fr active Pending
- 2016-02-29 AU AU2016224976A patent/AU2016224976A1/en not_active Abandoned
- 2016-02-29 WO PCT/US2016/020127 patent/WO2016138534A2/fr not_active Ceased
- 2016-03-01 TW TW112123102A patent/TW202403045A/zh unknown
- 2016-03-01 TW TW105106275A patent/TW201702378A/zh unknown
-
2017
- 2017-08-23 IL IL254112A patent/IL254112B/en active IP Right Grant
- 2017-08-25 MX MX2024010190A patent/MX2024010190A/es unknown
-
2020
- 2020-06-10 AU AU2020203825A patent/AU2020203825B2/en active Active
-
2021
- 2021-03-02 IL IL281199A patent/IL281199B/en unknown
- 2021-07-12 JP JP2021114792A patent/JP2021166543A/ja active Pending
-
2023
- 2023-07-14 JP JP2023115841A patent/JP2023129494A/ja active Pending
-
2024
- 2024-04-03 JP JP2024060083A patent/JP2024074908A/ja active Pending
- 2024-05-10 US US18/660,789 patent/US20250171783A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| TW202403045A (zh) | 2024-01-16 |
| US20250171783A1 (en) | 2025-05-29 |
| WO2016138534A3 (fr) | 2016-12-22 |
| TW201702378A (zh) | 2017-01-16 |
| JP2023129494A (ja) | 2023-09-14 |
| JP2021166543A (ja) | 2021-10-21 |
| EP3262056A2 (fr) | 2018-01-03 |
| US20180216111A1 (en) | 2018-08-02 |
| JP2018509143A (ja) | 2018-04-05 |
| IL254112A (en) | 2018-06-28 |
| BR112017018383B1 (pt) | 2023-04-25 |
| EP3262056A4 (fr) | 2018-09-19 |
| WO2016138534A2 (fr) | 2016-09-01 |
| IL281199A (en) | 2021-04-29 |
| CA2977528A1 (fr) | 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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