MX2022007491A - Acido nucleico antisentido que permite la omision de exones. - Google Patents
Acido nucleico antisentido que permite la omision de exones.Info
- Publication number
- MX2022007491A MX2022007491A MX2022007491A MX2022007491A MX2022007491A MX 2022007491 A MX2022007491 A MX 2022007491A MX 2022007491 A MX2022007491 A MX 2022007491A MX 2022007491 A MX2022007491 A MX 2022007491A MX 2022007491 A MX2022007491 A MX 2022007491A
- Authority
- MX
- Mexico
- Prior art keywords
- mrna
- target pre
- nucleic acid
- exons
- antisense nucleic
- Prior art date
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- 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)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (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)
Abstract
La presente especificación proporciona un oligómero antisentido capaz de provocar la omisión simultánea de una pluralidad de exones en pre-ARNm de interés, y una composición farmacéutica que comprende el oligómero. La presente especificación también proporciona un oligómero antisentido o una sal farmacéuticamente aceptable del mismo, o hidrato del mismo que provoca la omisión simultánea de dos o más exones numéricamente consecutivos del preARNm de interés, el oligómero antisentido que comprende una secuencia de bases complementaria a una secuencia de bases de una región que incluye la vecindad de un donador de cualquier intrón en el pre-ARNm de interés, o una región que incluye la vecindad de un aceptor de cualquier intrón en el pre-ARNm de interés, o una secuencia de bases parcial de la misma.
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 |
|---|---|
| MX2022007491A true MX2022007491A (es) | 2022-08-15 |
Family
ID=76478794
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2022007491A MX2022007491A (es) | 2019-12-19 | 2020-12-18 | Acido nucleico antisentido que permite la omision de exones. |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230045557A1 (es) |
| EP (1) | EP4079329A4 (es) |
| JP (1) | JPWO2021125311A1 (es) |
| KR (1) | KR20220118459A (es) |
| CN (1) | CN115151642A (es) |
| AU (1) | AU2020408773A1 (es) |
| BR (1) | BR112022012114A2 (es) |
| CA (1) | CA3165316A1 (es) |
| CO (1) | CO2022009983A2 (es) |
| IL (1) | IL293997A (es) |
| MX (1) | MX2022007491A (es) |
| TW (1) | TW202136511A (es) |
| WO (1) | WO2021125311A1 (es) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023171820A1 (ja) * | 2022-03-11 | 2023-09-14 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0962463B1 (en) | 1989-12-20 | 2002-07-10 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorus-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 |
| ES2566629T3 (es) | 2002-11-25 | 2016-04-14 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
| 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 |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| WO2010050801A1 (en) * | 2008-10-27 | 2010-05-06 | Prosensa Technologies B.V. | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna |
| CA2884340C (en) | 2007-11-15 | 2017-07-25 | Sarepta Therapeutics, Inc. | Method of synthesis of morpholino oligomers |
| 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 |
| CA3066050A1 (en) | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| KR20230137491A (ko) * | 2009-11-12 | 2023-10-04 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| RU2619184C2 (ru) | 2011-12-28 | 2017-05-12 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| EP2806900B1 (en) * | 2012-01-27 | 2021-12-15 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| ES2762881T3 (es) * | 2013-03-14 | 2020-05-26 | Sarepta Therapeutics Inc | Composiciones de salto de exón para el tratamiento de la distrofia muscular |
| WO2018129384A1 (en) * | 2017-01-06 | 2018-07-12 | Avidity Biosciences Llc | Nucleic acid-polypeptide compositions and methods of inducing exon skipping |
| CN111770757A (zh) * | 2017-09-22 | 2020-10-13 | 艾维迪提生物科学公司 | 诱导外显子跳读的核酸-多肽组合物和方法 |
| WO2019136216A1 (en) * | 2018-01-05 | 2019-07-11 | The Board Of Regents Of The University Of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| BR112020020670A2 (pt) * | 2018-04-12 | 2021-03-02 | Wave Life Sciences Ltd. | composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo |
| SG11202100934PA (en) * | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
-
2020
- 2020-12-18 CN CN202080097251.XA patent/CN115151642A/zh active Pending
- 2020-12-18 TW TW109145043A patent/TW202136511A/zh unknown
- 2020-12-18 EP EP20901000.8A patent/EP4079329A4/en active Pending
- 2020-12-18 US US17/785,565 patent/US20230045557A1/en active Pending
- 2020-12-18 BR BR112022012114A patent/BR112022012114A2/pt unknown
- 2020-12-18 AU AU2020408773A patent/AU2020408773A1/en active Pending
- 2020-12-18 JP JP2021565671A patent/JPWO2021125311A1/ja active Pending
- 2020-12-18 KR KR1020227023507A patent/KR20220118459A/ko unknown
- 2020-12-18 CA CA3165316A patent/CA3165316A1/en active Pending
- 2020-12-18 IL IL293997A patent/IL293997A/en unknown
- 2020-12-18 MX MX2022007491A patent/MX2022007491A/es unknown
- 2020-12-18 WO PCT/JP2020/047340 patent/WO2021125311A1/ja active Application Filing
-
2022
- 2022-07-15 CO CONC2022/0009983A patent/CO2022009983A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JPWO2021125311A1 (es) | 2021-06-24 |
| EP4079329A4 (en) | 2024-04-17 |
| AU2020408773A1 (en) | 2022-07-14 |
| BR112022012114A2 (pt) | 2022-09-06 |
| EP4079329A1 (en) | 2022-10-26 |
| CN115151642A (zh) | 2022-10-04 |
| CA3165316A1 (en) | 2021-06-24 |
| WO2021125311A1 (ja) | 2021-06-24 |
| TW202136511A (zh) | 2021-10-01 |
| KR20220118459A (ko) | 2022-08-25 |
| CO2022009983A2 (es) | 2022-07-19 |
| IL293997A (en) | 2022-08-01 |
| US20230045557A1 (en) | 2023-02-09 |
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