CO2022009983A2 - Ácido nucleico antisentido que permite la omisión de exones - Google Patents
Ácido nucleico antisentido que permite la omisión de exonesInfo
- Publication number
- CO2022009983A2 CO2022009983A2 CONC2022/0009983A CO2022009983A CO2022009983A2 CO 2022009983 A2 CO2022009983 A2 CO 2022009983A2 CO 2022009983 A CO2022009983 A CO 2022009983A CO 2022009983 A2 CO2022009983 A2 CO 2022009983A2
- Authority
- CO
- Colombia
- Prior art keywords
- mrna
- interest
- nucleic acid
- antisense nucleic
- exon skipping
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/31—Chemical structure of the backbone
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- 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
-
- 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)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019229763 | 2019-12-19 | ||
| PCT/JP2020/047340 WO2021125311A1 (ja) | 2019-12-19 | 2020-12-18 | エクソンスキッピングを可能にするアンチセンス核酸 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2022009983A2 true CO2022009983A2 (es) | 2022-07-19 |
Family
ID=76478794
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2022/0009983A CO2022009983A2 (es) | 2019-12-19 | 2022-07-15 | Ácido nucleico antisentido que permite la omisión de exones |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20230045557A1 (https=) |
| EP (1) | EP4079329A4 (https=) |
| JP (1) | JP7842395B2 (https=) |
| KR (1) | KR20220118459A (https=) |
| CN (1) | CN115151642A (https=) |
| AU (1) | AU2020408773A1 (https=) |
| BR (1) | BR112022012114A2 (https=) |
| CA (1) | CA3165316A1 (https=) |
| CO (1) | CO2022009983A2 (https=) |
| IL (1) | IL293997A (https=) |
| MX (1) | MX2022007491A (https=) |
| PH (1) | PH12022551438A1 (https=) |
| TW (1) | TW202136511A (https=) |
| WO (1) | WO2021125311A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4363578A1 (en) * | 2021-07-02 | 2024-05-08 | Aarhus Universitet | Methods of treating alzheimer's disease |
| JP2025079347A (ja) * | 2022-03-11 | 2025-05-22 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
| EP4680250A1 (en) * | 2023-03-17 | 2026-01-21 | The Regents of the University of California | Factor viii splice-modulating antisense oligonucleotides and methods of use |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| EP2392660A3 (en) | 2002-11-25 | 2012-03-28 | Masafumi Matsuo | ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor |
| AU2003225410A1 (en) * | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| AU2009310557B2 (en) * | 2008-10-27 | 2014-09-11 | Academisch Ziekenhuis Leiden | Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CA2861247C (en) | 2011-12-28 | 2021-11-16 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
| NZ627896A (en) * | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| 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 |
| DK3118311T3 (en) * | 2014-03-12 | 2019-03-11 | Nippon Shinyaku Co Ltd | Antisense nucleic acid |
| HUE050061T2 (hu) * | 2015-09-15 | 2020-12-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| MX2019008199A (es) * | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| MX2020003130A (es) * | 2017-09-22 | 2020-09-25 | Avidity Biosciences Inc | Composiciones y metodos de induccion de salto de exon en acidos nucleicos-polipeptidos. |
| EP3735462A1 (en) * | 2018-01-05 | 2020-11-11 | The Board of Regents of The University of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| SG11202009877XA (en) * | 2018-04-12 | 2020-11-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
| CA3108282A1 (en) * | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN114901823A (zh) * | 2019-12-26 | 2022-08-12 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| IL295967A (en) * | 2020-02-28 | 2022-10-01 | Nippon Shinyaku Co Ltd | Antisense nucleic acids for splicing on exon 51 |
-
2020
- 2020-12-18 IL IL293997A patent/IL293997A/en unknown
- 2020-12-18 AU AU2020408773A patent/AU2020408773A1/en active Pending
- 2020-12-18 PH PH1/2022/551438A patent/PH12022551438A1/en unknown
- 2020-12-18 US US17/785,565 patent/US20230045557A1/en active Pending
- 2020-12-18 EP EP20901000.8A patent/EP4079329A4/en active Pending
- 2020-12-18 KR KR1020227023507A patent/KR20220118459A/ko active Pending
- 2020-12-18 WO PCT/JP2020/047340 patent/WO2021125311A1/ja not_active Ceased
- 2020-12-18 BR BR112022012114A patent/BR112022012114A2/pt unknown
- 2020-12-18 TW TW109145043A patent/TW202136511A/zh unknown
- 2020-12-18 JP JP2021565671A patent/JP7842395B2/ja active Active
- 2020-12-18 CN CN202080097251.XA patent/CN115151642A/zh active Pending
- 2020-12-18 CA CA3165316A patent/CA3165316A1/en active Pending
- 2020-12-18 MX MX2022007491A patent/MX2022007491A/es unknown
-
2022
- 2022-07-15 CO CONC2022/0009983A patent/CO2022009983A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN115151642A (zh) | 2022-10-04 |
| PH12022551438A1 (en) | 2023-11-20 |
| JP7842395B2 (ja) | 2026-04-08 |
| CA3165316A1 (en) | 2021-06-24 |
| AU2020408773A1 (en) | 2022-07-14 |
| MX2022007491A (es) | 2022-08-15 |
| JPWO2021125311A1 (https=) | 2021-06-24 |
| IL293997A (en) | 2022-08-01 |
| KR20220118459A (ko) | 2022-08-25 |
| WO2021125311A1 (ja) | 2021-06-24 |
| EP4079329A4 (en) | 2024-04-17 |
| TW202136511A (zh) | 2021-10-01 |
| US20230045557A1 (en) | 2023-02-09 |
| EP4079329A1 (en) | 2022-10-26 |
| BR112022012114A2 (pt) | 2022-09-06 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CO2022009983A2 (es) | Ácido nucleico antisentido que permite la omisión de exones | |
| CO2019007839A2 (es) | Composiciones y métodos para inhibir la acción de la arginasa | |
| MX2016016526A (es) | Acidos nucleicos antisentido. | |
| CL2018001899A1 (es) | Salicilamidas de espiroheptanos y compuestos relacionados como inhibidores de cinasa rho (rock) | |
| CL2018003352A1 (es) | Compuestos de carfilzomib pegilados. | |
| MX2017011013A (es) | Terapia de oligonucleotidos para amaurosis congenita de leber. | |
| CL2020000215A1 (es) | Compuestos y composiciones para tratar afecciones asociadas con la actividad nlrp. | |
| CY1122821T1 (el) | Παραγωγα 1,3-βενζοδιοξολιου ως αναστολεις εζη1 ή/και εζη2 | |
| EA201890864A1 (ru) | Композиции и способы для ингибирования генной экспрессии лпа | |
| CL2020002698A1 (es) | Moduladores de enzimas modificadoras de metilo, composiciones y usos de estos | |
| CY1123119T1 (el) | Αντινοηματικο νουκλεϊκο οξυ | |
| MX387088B (es) | Complemento comprimible con estructuras de soporte intermedias. | |
| CO2017004525A2 (es) | Dihidropirrolopiridinas inhibidoras de ror-gamma | |
| MX381890B (es) | Combinaciones de inhibidores de desmetilasa-1 especifica de lisina (lsd1) para uso en el tratamiento de tumores solidos | |
| MX2017002656A (es) | Compuestos que inhiben la proteína mcl-1. | |
| MX2017000143A (es) | Composiciones enriquecidas estereoquimicamente para administracion de acidos nucleicos. | |
| MX2018000777A (es) | Compuestos de quinazolina sustituido y su uso como inhibidores de proteinas kras, hras y/o nras mutantes g12c. | |
| EA201991450A1 (ru) | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии | |
| RU2016150116A (ru) | Ингибитор пролиферации раковых стволовых клеток | |
| SV2017005354A (es) | Derivados de quinolizinona como inhibidores de pi3k | |
| MX389692B (es) | Composiciones y métodos para promover el crecimiento del cabello con inhibidores de mpc. | |
| BR112022012622A2 (pt) | Ácido nucleico antissenso que induz o salto do éxon 50 | |
| WO2017087947A3 (en) | Method of treatment of follicular lymphoma with a btk inhibitor | |
| CL2023003606A1 (es) | Oligómeros que omiten exones para la distrofia muscular div 202100203 | |
| EA202191601A1 (ru) | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |