RU2017140065A - Композиции нуклеиновой кислоты-катионного полимера и способы их получения и применения - Google Patents
Композиции нуклеиновой кислоты-катионного полимера и способы их получения и применения Download PDFInfo
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- RU2017140065A RU2017140065A RU2017140065A RU2017140065A RU2017140065A RU 2017140065 A RU2017140065 A RU 2017140065A RU 2017140065 A RU2017140065 A RU 2017140065A RU 2017140065 A RU2017140065 A RU 2017140065A RU 2017140065 A RU2017140065 A RU 2017140065A
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- carbohydrate
- nucleic acid
- poly
- cationic polymer
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- 238000004108 freeze drying Methods 0.000 claims 2
- 125000000524 functional group Chemical group 0.000 claims 2
- 229930182830 galactose Natural products 0.000 claims 2
- 125000002791 glucosyl group Chemical group C1([C@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 2
- 229940096919 glycogen Drugs 0.000 claims 2
- 229960003786 inosine Drugs 0.000 claims 2
- 239000000594 mannitol Substances 0.000 claims 2
- 235000010355 mannitol Nutrition 0.000 claims 2
- 108020001162 nitroreductase Proteins 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- 229920000724 poly(L-arginine) polymer Polymers 0.000 claims 2
- 229920000083 poly(allylamine) Polymers 0.000 claims 2
- 229920001308 poly(aminoacid) Polymers 0.000 claims 2
- 229920002643 polyglutamic acid Polymers 0.000 claims 2
- 108010055896 polyornithine Proteins 0.000 claims 2
- 229920002717 polyvinylpyridine Polymers 0.000 claims 2
- 235000018102 proteins Nutrition 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108091092562 ribozyme Proteins 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 239000000600 sorbitol Substances 0.000 claims 2
- UQZIYBXSHAGNOE-XNSRJBNMSA-N stachyose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO[C@@H]3[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O3)O)O2)O)O1 UQZIYBXSHAGNOE-XNSRJBNMSA-N 0.000 claims 2
- 239000008107 starch Substances 0.000 claims 2
- 235000019698 starch Nutrition 0.000 claims 2
- 150000004043 trisaccharides Chemical class 0.000 claims 2
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 2
- MQJSSLBGAQJNER-UHFFFAOYSA-N 5-(methylaminomethyl)-1h-pyrimidine-2,4-dione Chemical compound CNCC1=CNC(=O)NC1=O MQJSSLBGAQJNER-UHFFFAOYSA-N 0.000 claims 1
- KELXHQACBIUYSE-UHFFFAOYSA-N 5-methoxy-1h-pyrimidine-2,4-dione Chemical compound COC1=CNC(=O)NC1=O KELXHQACBIUYSE-UHFFFAOYSA-N 0.000 claims 1
- ZLAQATDNGLKIEV-UHFFFAOYSA-N 5-methyl-2-sulfanylidene-1h-pyrimidin-4-one Chemical compound CC1=CNC(=S)NC1=O ZLAQATDNGLKIEV-UHFFFAOYSA-N 0.000 claims 1
- BYPCVKNBGDKXLB-UHFFFAOYSA-N 6-(aminomethyl)-5-methyl-1h-pyrimidine-2,4-dione Chemical compound CC1=C(CN)NC(=O)NC1=O BYPCVKNBGDKXLB-UHFFFAOYSA-N 0.000 claims 1
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- 108050006400 Cyclin Proteins 0.000 claims 1
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims 1
- 229930182816 L-glutamine Natural products 0.000 claims 1
- 102000001253 Protein Kinase Human genes 0.000 claims 1
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims 1
- 230000002759 chromosomal effect Effects 0.000 claims 1
- 206010013023 diphtheria Diseases 0.000 claims 1
- 108060006633 protein kinase Proteins 0.000 claims 1
- 238000003756 stirring Methods 0.000 claims 1
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- 229940104230 thymidine Drugs 0.000 claims 1
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- 231100000765 toxin Toxicity 0.000 claims 1
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
- A61K48/0008—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
- A61K48/0041—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 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
- A61K48/005—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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- 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
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Dermatology (AREA)
- Manufacturing & Machinery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| IL23864315 | 2015-05-05 | ||
| IL238643 | 2015-05-05 | ||
| PCT/IL2016/050477 WO2016178233A1 (en) | 2015-05-05 | 2016-05-05 | Nucleic acid-cationic polymer compositions and methods of making and using the same |
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| RU2017140065A true RU2017140065A (ru) | 2019-06-05 |
| RU2017140065A3 RU2017140065A3 (enExample) | 2019-09-27 |
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| US (2) | US20180154023A1 (enExample) |
| EP (1) | EP3291799A1 (enExample) |
| JP (2) | JP2018515479A (enExample) |
| KR (1) | KR20180015145A (enExample) |
| CN (1) | CN107735079A (enExample) |
| AU (1) | AU2016258326A1 (enExample) |
| BR (1) | BR112017023788A2 (enExample) |
| CA (1) | CA2984879A1 (enExample) |
| IL (1) | IL255254A0 (enExample) |
| MX (1) | MX2017014060A (enExample) |
| PH (1) | PH12017502216A1 (enExample) |
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| SG (2) | SG11201708760PA (enExample) |
| WO (1) | WO2016178233A1 (enExample) |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| JP2018515479A (ja) * | 2015-05-05 | 2018-06-14 | イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドYissum Research Development Company Of The Hebrew Universty Of Jerusalem Ltd. | 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法 |
| DE102017003004A1 (de) | 2017-03-23 | 2018-09-27 | Friedrich-Schiller-Universität Jena | Kationische Polymere mit D-Fructose-Substituenten |
| US10849921B2 (en) * | 2017-05-17 | 2020-12-01 | Bioncotech Therapeutics Sl | Pharmaceutical composition comprising particles comprising a complex of a double-stranded polyribonucleotide and a polyalkyleneimine |
| EP3924463A1 (en) | 2019-02-11 | 2021-12-22 | Excellgene SA | Novel eukaryotic cell transfection systems and related methods |
| CN111363761A (zh) * | 2020-03-17 | 2020-07-03 | 苏州吉恒基因科技有限公司 | 利用阳离子多聚体dna复合物促进aav介导的基因表达方法 |
| CA3181339A1 (en) * | 2020-04-27 | 2021-11-04 | Juno Therapeutics, Inc. | Stabilization of polyethyleneimine-deoxyribonucleic acid complex size and activity |
| TW202228727A (zh) * | 2020-10-01 | 2022-08-01 | 德商拜恩迪克公司 | 適用於治療之微脂體rna調配物之製備及儲存 |
| WO2022249180A1 (en) | 2021-05-25 | 2022-12-01 | Ramot At Tel-Aviv University Ltd. | Compositions comprising extracellular vesicles and an active agent and uses thereof |
| CN113501889A (zh) * | 2021-07-06 | 2021-10-15 | 郑州大学 | 一种三七多糖阳离子衍生物的制备方法及其应用 |
| CN116549626A (zh) * | 2022-01-27 | 2023-08-08 | 深圳瑞吉生物科技有限公司 | 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用 |
| CN114762731B (zh) * | 2022-05-16 | 2024-08-23 | 常州大学 | 一种延长基因药物在温和条件下储存时间的方法 |
| CN116036043A (zh) * | 2023-01-10 | 2023-05-02 | 深圳市乐土生物医药有限公司 | 一种核酸药物制剂、冻干粉及制备方法 |
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| US5116742A (en) | 1986-12-03 | 1992-05-26 | University Patents, Inc. | RNA ribozyme restriction endoribonucleases and methods |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| FR2722506B1 (fr) | 1994-07-13 | 1996-08-14 | Rhone Poulenc Rorer Sa | Composition contenant des acides nucleiques, preparation et utilisations |
| US6087164A (en) * | 1997-10-03 | 2000-07-11 | Yissum Research Development Company Of The Hebrew University Of Jerusalem | Methods and compositions for inducing tumor-specific cytotoxicity |
| US20040142474A1 (en) * | 2000-09-14 | 2004-07-22 | Expression Genetics, Inc. | Novel cationic lipopolymer as a biocompatible gene delivery agent |
| WO2007007317A1 (en) | 2005-07-07 | 2007-01-18 | Yissum Research Development Company Of The Hebrew University Of Jerusalem | Nucleic acid agents for downregulating h19, and methods of using same |
| JP2009508516A (ja) | 2005-09-22 | 2009-03-05 | イッサム リサーチ ディベロプメント カンパニー オブ ザ ヘブリュー ユニバーシティ オブ エルサレム | 核酸構築物、薬剤組成物、及び癌治療のためのその使用方法 |
| CA2675967A1 (en) | 2007-01-16 | 2008-07-24 | Yissum Research Development Company Of The Hebrew University Of Jerusale M | Nucleic acid constructs and methods for specific silencing of h19 |
| US20090042825A1 (en) * | 2007-08-06 | 2009-02-12 | Majed Matar | Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer |
| CN101820864A (zh) * | 2007-08-06 | 2010-09-01 | 艾根股份有限公司 | 核酸-脂聚合物组合物 |
| BRPI0818937B8 (pt) | 2007-10-25 | 2021-05-25 | Yissum Res Dev Co Of Hebrew Univ Jerusalem Ltd | constructos contendo múltiplos cassetes de expressão para terapia de câncer |
| WO2010028093A2 (en) * | 2008-09-03 | 2010-03-11 | Senesco Technologies, Inc. | Use of a truncated elf-5a1 polynucleotide to induce apoptosis in cancer cells |
| DE102009006606A1 (de) * | 2009-01-29 | 2010-08-05 | Philipps-Universität Marburg | Nicht-virales Transfektionsmittel |
| JP2018515479A (ja) * | 2015-05-05 | 2018-06-14 | イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドYissum Research Development Company Of The Hebrew Universty Of Jerusalem Ltd. | 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法 |
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2016
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- 2016-05-05 RU RU2017140065A patent/RU2017140065A/ru not_active Application Discontinuation
- 2016-05-05 BR BR112017023788A patent/BR112017023788A2/pt not_active Application Discontinuation
- 2016-05-05 CA CA2984879A patent/CA2984879A1/en not_active Abandoned
- 2016-05-05 WO PCT/IL2016/050477 patent/WO2016178233A1/en not_active Ceased
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2020
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2021
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| EP3291799A1 (en) | 2018-03-14 |
| US20200138977A1 (en) | 2020-05-07 |
| US20180154023A1 (en) | 2018-06-07 |
| BR112017023788A2 (pt) | 2018-10-16 |
| KR20180015145A (ko) | 2018-02-12 |
| CN107735079A (zh) | 2018-02-23 |
| MX2017014060A (es) | 2018-07-06 |
| CA2984879A1 (en) | 2016-11-10 |
| JP2021130671A (ja) | 2021-09-09 |
| AU2016258326A1 (en) | 2017-11-23 |
| WO2016178233A1 (en) | 2016-11-10 |
| RU2017140065A3 (enExample) | 2019-09-27 |
| SG11201708760PA (en) | 2017-11-29 |
| PH12017502216A1 (en) | 2018-06-11 |
| JP2018515479A (ja) | 2018-06-14 |
| SG10201909209RA (en) | 2019-11-28 |
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| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20200123 |