JP6113737B2 - 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 - Google Patents
修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 Download PDFInfo
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- JP6113737B2 JP6113737B2 JP2014534647A JP2014534647A JP6113737B2 JP 6113737 B2 JP6113737 B2 JP 6113737B2 JP 2014534647 A JP2014534647 A JP 2014534647A JP 2014534647 A JP2014534647 A JP 2014534647A JP 6113737 B2 JP6113737 B2 JP 6113737B2
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Classifications
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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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/19—Cytokines; Lymphokines; Interferons
- A61K38/193—Colony stimulating factors [CSF]
-
- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- 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
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
-
- 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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/53—Colony-stimulating factor [CSF]
- C07K14/535—Granulocyte CSF; Granulocyte-macrophage CSF
-
- 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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Dermatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161542533P | 2011-10-03 | 2011-10-03 | |
| US61/542,533 | 2011-10-03 | ||
| PCT/US2012/058519 WO2013052523A1 (en) | 2011-10-03 | 2012-10-03 | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017049969A Division JP6388977B2 (ja) | 2011-10-03 | 2017-03-15 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014530601A JP2014530601A (ja) | 2014-11-20 |
| JP2014530601A5 JP2014530601A5 (enExample) | 2015-11-26 |
| JP6113737B2 true JP6113737B2 (ja) | 2017-04-12 |
Family
ID=48044115
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014534647A Active JP6113737B2 (ja) | 2011-10-03 | 2012-10-03 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2017049969A Active JP6388977B2 (ja) | 2011-10-03 | 2017-03-15 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2018152801A Active JP6574032B2 (ja) | 2011-10-03 | 2018-08-15 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2019148703A Active JP7019639B2 (ja) | 2011-10-03 | 2019-08-14 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2022014572A Pending JP2022060269A (ja) | 2011-10-03 | 2022-02-02 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2024018741A Pending JP2024036694A (ja) | 2011-10-03 | 2024-02-09 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017049969A Active JP6388977B2 (ja) | 2011-10-03 | 2017-03-15 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2018152801A Active JP6574032B2 (ja) | 2011-10-03 | 2018-08-15 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2019148703A Active JP7019639B2 (ja) | 2011-10-03 | 2019-08-14 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2022014572A Pending JP2022060269A (ja) | 2011-10-03 | 2022-02-02 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| JP2024018741A Pending JP2024036694A (ja) | 2011-10-03 | 2024-02-09 | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
Country Status (25)
| Country | Link |
|---|---|
| US (5) | US9428535B2 (enExample) |
| EP (4) | EP2763701B1 (enExample) |
| JP (6) | JP6113737B2 (enExample) |
| KR (2) | KR102014061B1 (enExample) |
| CN (2) | CN103974724B (enExample) |
| AU (5) | AU2012318752B2 (enExample) |
| BR (1) | BR112014007852B1 (enExample) |
| CA (1) | CA2850624A1 (enExample) |
| CY (1) | CY1125029T1 (enExample) |
| DE (1) | DE19216461T1 (enExample) |
| DK (1) | DK3682905T3 (enExample) |
| ES (1) | ES2911677T3 (enExample) |
| HR (1) | HRP20220250T1 (enExample) |
| HU (1) | HUE057725T2 (enExample) |
| IL (2) | IL231808B (enExample) |
| LT (1) | LT3682905T (enExample) |
| MX (1) | MX354267B (enExample) |
| PL (1) | PL3682905T3 (enExample) |
| PT (1) | PT3682905T (enExample) |
| RS (1) | RS62993B1 (enExample) |
| RU (2) | RU2648950C2 (enExample) |
| SG (2) | SG10201602654SA (enExample) |
| SI (1) | SI3682905T1 (enExample) |
| SM (1) | SMT202200229T1 (enExample) |
| WO (1) | WO2013052523A1 (enExample) |
Families Citing this family (384)
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| DE10229872A1 (de) | 2002-07-03 | 2004-01-29 | Curevac Gmbh | Immunstimulation durch chemisch modifizierte RNA |
| DE10347710B4 (de) | 2003-10-14 | 2006-03-30 | Johannes-Gutenberg-Universität Mainz | Rekombinante Impfstoffe und deren Verwendung |
| DE102005046490A1 (de) | 2005-09-28 | 2007-03-29 | Johannes-Gutenberg-Universität Mainz | Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen |
| PT3243526T (pt) | 2010-07-06 | 2020-03-04 | Glaxosmithkline Biologicals Sa | Distribuição de arn para despoletar múltiplas vias imunitárias |
| US10487332B2 (en) | 2010-07-06 | 2019-11-26 | Glaxosmithkline Biologicals Sa | Immunisation of large mammals with low doses of RNA |
| CN103153284B (zh) | 2010-07-06 | 2015-11-25 | 诺华股份有限公司 | 含有其pKa值有利于RNA递送的脂质的脂质体 |
| CA2807552A1 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| JP5911870B2 (ja) | 2010-08-31 | 2016-04-27 | ノバルティス アーゲー | 免疫原をコードするｒｎａの送達のためのｐｅｇ化リポソーム |
| EP2857499A1 (en) | 2010-10-01 | 2015-04-08 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| KR102266691B1 (ko) | 2010-10-11 | 2021-06-23 | 노파르티스 아게 | 항원 전달 플랫폼 |
| CA2831613A1 (en) | 2011-03-31 | 2012-10-04 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| LT2714071T (lt) | 2011-05-24 | 2019-10-10 | Biontech Rna Pharmaceuticals Gmbh | Individualizuotos vakcinos nuo vėžio |
| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| JP2014522842A (ja) | 2011-07-06 | 2014-09-08 | ノバルティス アーゲー | 免疫原性組み合わせ組成物およびその使用 |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| PT3682905T (pt) | 2011-10-03 | 2022-04-07 | Modernatx Inc | Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações |
| US8497124B2 (en) | 2011-12-05 | 2013-07-30 | Factor Bioscience Inc. | Methods and products for reprogramming cells to a less differentiated state |
| EP3260140B1 (en) | 2011-12-05 | 2021-02-03 | Factor Bioscience Inc. | Methods and products for transfecting cells |
| ES2923757T3 (es) | 2011-12-16 | 2022-09-30 | Modernatx Inc | Composiciones de ARNm modificado |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| US9890364B2 (en) | 2012-05-29 | 2018-02-13 | The General Hospital Corporation | TAL-Tet1 fusion proteins and methods of use thereof |
| CN104603271B (zh) | 2012-07-03 | 2019-07-19 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
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| CA2890110C (en) | 2012-11-01 | 2023-05-02 | Factor Bioscience Inc. | Methods and products for expressing proteins in cells |
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| AU2013351542B2 (en) | 2012-11-28 | 2018-08-09 | BioNTech SE | Individualized vaccines for cancer |
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| US20160024181A1 (en) | 2013-03-13 | 2016-01-28 | Moderna Therapeutics, Inc. | Long-lived polynucleotide molecules |
| KR102311614B1 (ko) | 2013-03-14 | 2021-10-08 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
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