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
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
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 (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) | TW201702378A (fr) |
WO (1) | WO2016138534A2 (fr) |
Families Citing this family (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3041935A1 (fr) | 2013-09-05 | 2016-07-13 | Sage Therapeutics, Inc. | Inclusion de l'exon 2 induite par antisens dans une alpha-glucosidase acide |
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 |
KR20240035901A (ko) | 2015-05-19 | 2024-03-18 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
KR20220105174A (ko) | 2015-10-09 | 2022-07-26 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
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 |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
JP2018538302A (ja) | 2015-12-15 | 2018-12-27 | サレプタ セラピューティクス, インコーポレイテッド | ペプチドオリゴヌクレオチド複合体 |
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. |
SG10202108375XA (en) | 2017-08-25 | 2021-09-29 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
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 |
KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
US20230287410A1 (en) | 2020-05-11 | 2023-09-14 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
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 |
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)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
CA2799501C (fr) * | 2010-05-28 | 2022-02-15 | Sarepta Therapeutics, Inc. | Analogues oligonucleotidiques ayant des liaisons modifiees entre sous-unites et/ou des groupes terminaux modifies |
KR102183273B1 (ko) * | 2011-05-05 | 2020-11-27 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 펩타이드 올리고뉴클레오타이드 접합체 |
US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
EA035030B1 (ru) * | 2011-11-18 | 2020-04-20 | Сарепта Терапьютикс, Инк. | Модифицированные морфолиновые аналоги олигонуклеотидов |
BR122020016865B1 (pt) * | 2013-03-14 | 2022-12-27 | Sarepta Therapeutics, Inc. | Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd) |
EP3041935A1 (fr) * | 2013-09-05 | 2016-07-13 | Sage Therapeutics, Inc. | Inclusion de l'exon 2 induite par antisens dans une alpha-glucosidase acide |
EP3143141B1 (fr) * | 2014-05-16 | 2019-10-02 | Oregon State University | Composés antisens antibactériens et procédés associés |
US10308940B2 (en) * | 2014-06-10 | 2019-06-04 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of Pompe disease |
-
2016
- 2016-02-29 WO PCT/US2016/020127 patent/WO2016138534A2/fr active Application Filing
- 2016-02-29 US US15/553,911 patent/US20180216111A1/en not_active Abandoned
- 2016-02-29 MX MX2017011004A patent/MX2017011004A/es unknown
- 2016-02-29 AU AU2016224976A patent/AU2016224976A1/en not_active Abandoned
- 2016-02-29 EP EP16756555.5A patent/EP3262056A4/fr active Pending
- 2016-02-29 MA MA041759A patent/MA41759A/fr unknown
- 2016-02-29 CA CA2977528A patent/CA2977528A1/fr active Pending
- 2016-02-29 JP JP2017545273A patent/JP2018509143A/ja not_active Ceased
- 2016-02-29 BR BR112017018383-8A patent/BR112017018383B1/pt active IP Right Grant
- 2016-03-01 TW TW105106275A patent/TW201702378A/zh unknown
- 2016-03-01 TW TW112123102A patent/TW202403045A/zh unknown
-
2017
- 2017-08-23 IL IL254112A patent/IL254112B/en active IP Right Grant
-
2018
- 2018-07-03 HK HK18108560.3A patent/HK1249106A1/zh 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
Also Published As
Publication number | Publication date |
---|---|
IL281199A (en) | 2021-04-29 |
MX2017011004A (es) | 2018-02-09 |
TW202403045A (zh) | 2024-01-16 |
JP2023129494A (ja) | 2023-09-14 |
AU2016224976A1 (en) | 2017-09-14 |
IL254112A (en) | 2018-06-28 |
EP3262056A2 (fr) | 2018-01-03 |
AU2020203825B2 (en) | 2021-08-05 |
TW201702378A (zh) | 2017-01-16 |
AU2020203825A1 (en) | 2020-07-02 |
BR112017018383B1 (pt) | 2023-04-25 |
US20180216111A1 (en) | 2018-08-02 |
HK1249106A1 (zh) | 2018-10-26 |
JP2018509143A (ja) | 2018-04-05 |
IL254112B (en) | 2021-04-29 |
CA2977528A1 (fr) | 2016-09-01 |
JP2021166543A (ja) | 2021-10-21 |
IL281199B (en) | 2022-05-01 |
EP3262056A4 (fr) | 2018-09-19 |
WO2016138534A3 (fr) | 2016-12-22 |
BR112017018383A2 (pt) | 2018-09-04 |
WO2016138534A2 (fr) | 2016-09-01 |
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