KR20220019676A - 선택적 인트론의 스플라이싱을 조절하기 위한 방법 및 조성물 - Google Patents
선택적 인트론의 스플라이싱을 조절하기 위한 방법 및 조성물 Download PDFInfo
- Publication number
- KR20220019676A KR20220019676A KR1020217038370A KR20217038370A KR20220019676A KR 20220019676 A KR20220019676 A KR 20220019676A KR 1020217038370 A KR1020217038370 A KR 1020217038370A KR 20217038370 A KR20217038370 A KR 20217038370A KR 20220019676 A KR20220019676 A KR 20220019676A
- Authority
- KR
- South Korea
- Prior art keywords
- mrna
- aic
- intron
- target protein
- protein
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
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Classifications
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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
- 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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- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
- C12N15/1138—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 receptors or cell surface proteins
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/322—2'-R Modification
-
- 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)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Neurosurgery (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Neurology (AREA)
- Obesity (AREA)
- Ophthalmology & Optometry (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962839572P | 2019-04-26 | 2019-04-26 | |
| US62/839,572 | 2019-04-26 | ||
| PCT/US2020/029953 WO2020219977A1 (en) | 2019-04-26 | 2020-04-24 | Methods and compositions for modulating splicing of alternative introns |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20220019676A true KR20220019676A (ko) | 2022-02-17 |
Family
ID=72941393
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217038370A Pending KR20220019676A (ko) | 2019-04-26 | 2020-04-24 | 선택적 인트론의 스플라이싱을 조절하기 위한 방법 및 조성물 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220162599A1 (https=) |
| EP (1) | EP3959313A4 (https=) |
| JP (2) | JP2022532998A (https=) |
| KR (1) | KR20220019676A (https=) |
| CN (1) | CN114127286A (https=) |
| AU (1) | AU2020263581A1 (https=) |
| BR (1) | BR112021021384A2 (https=) |
| CA (1) | CA3134947A1 (https=) |
| MX (1) | MX2021013081A (https=) |
| WO (1) | WO2020219977A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2023348346A1 (en) * | 2022-09-30 | 2025-03-27 | Contera Pharma A/S | Antisense oligonucleotides for the treatment of canavan disease |
| CN121059628A (zh) * | 2024-06-03 | 2025-12-05 | 时夕(广州)生物科技有限公司 | 调控RNA剪接的向导agRNA |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060058253A1 (en) * | 2002-08-12 | 2006-03-16 | Benoit Chabot | Methods to reprogram splice site selection in pre-messenger rnas |
| EP2555778A4 (en) * | 2010-04-06 | 2014-05-21 | Alnylam Pharmaceuticals Inc | COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION |
| AU2015206603B9 (en) * | 2014-01-14 | 2019-07-18 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for identification, assessment, prevention, and treatment of melanoma using PD-L1 isoforms |
| AU2015327836B2 (en) * | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| WO2017100587A1 (en) * | 2015-12-09 | 2017-06-15 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof |
| 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 |
| RU2747822C2 (ru) * | 2016-03-14 | 2021-05-14 | Ф. Хоффманн-Ля Рош Аг | Олигонуклеотиды для понижения экспрессии pd-l1 |
| SG10201705285SA (en) * | 2017-06-27 | 2019-01-30 | Agency Science Tech & Res | Antisense oligonucleotides |
-
2020
- 2020-04-24 MX MX2021013081A patent/MX2021013081A/es unknown
- 2020-04-24 JP JP2021563623A patent/JP2022532998A/ja active Pending
- 2020-04-24 CA CA3134947A patent/CA3134947A1/en active Pending
- 2020-04-24 CN CN202080040348.7A patent/CN114127286A/zh active Pending
- 2020-04-24 BR BR112021021384A patent/BR112021021384A2/pt unknown
- 2020-04-24 EP EP20795450.4A patent/EP3959313A4/en active Pending
- 2020-04-24 KR KR1020217038370A patent/KR20220019676A/ko active Pending
- 2020-04-24 WO PCT/US2020/029953 patent/WO2020219977A1/en not_active Ceased
- 2020-04-24 AU AU2020263581A patent/AU2020263581A1/en active Pending
-
2021
- 2021-11-03 US US17/518,337 patent/US20220162599A1/en active Pending
-
2025
- 2025-05-27 JP JP2025087686A patent/JP2025128180A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3959313A4 (en) | 2023-09-27 |
| BR112021021384A2 (pt) | 2021-12-21 |
| US20220162599A1 (en) | 2022-05-26 |
| CN114127286A (zh) | 2022-03-01 |
| AU2020263581A1 (en) | 2021-12-16 |
| MX2021013081A (es) | 2021-12-10 |
| WO2020219977A1 (en) | 2020-10-29 |
| EP3959313A1 (en) | 2022-03-02 |
| JP2022532998A (ja) | 2022-07-21 |
| JP2025128180A (ja) | 2025-09-02 |
| CA3134947A1 (en) | 2020-10-29 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
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| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
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| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
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| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |
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| PA0201 | Request for examination |
St.27 status event code: A-1-2-D10-D11-exm-PA0201 |
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| D21 | Rejection of application intended |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| PE0902 | Notice of grounds for rejection |
St.27 status event code: A-1-2-D10-D21-exm-PE0902 |
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| E13 | Pre-grant limitation requested |
Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| E13-X000 | Pre-grant limitation requested |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| P11-X000 | Amendment of application requested |
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