RU2014117690A - Композиции и способы для сайленсинга альдегид-дегидрогеназы - Google Patents
Композиции и способы для сайленсинга альдегид-дегидрогеназы Download PDFInfo
- Publication number
- RU2014117690A RU2014117690A RU2014117690/10A RU2014117690A RU2014117690A RU 2014117690 A RU2014117690 A RU 2014117690A RU 2014117690/10 A RU2014117690/10 A RU 2014117690/10A RU 2014117690 A RU2014117690 A RU 2014117690A RU 2014117690 A RU2014117690 A RU 2014117690A
- Authority
- RU
- Russia
- Prior art keywords
- lipid
- particle
- nucleic acid
- interfering rna
- composition
- Prior art date
Links
Classifications
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
- A61P25/32—Alcohol-abuse
-
- 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
- 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
- C12N15/1137—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 enzymes
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y102/00—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
- C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
- C12Y102/01003—Aldehyde dehydrogenase (NAD+) (1.2.1.3)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Addiction (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dispersion Chemistry (AREA)
- Psychiatry (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161543700P | 2011-10-05 | 2011-10-05 | |
| US61/543,700 | 2011-10-05 | ||
| US201261599238P | 2012-02-15 | 2012-02-15 | |
| US61/599,238 | 2012-02-15 | ||
| PCT/US2012/058770 WO2013052677A1 (en) | 2011-10-05 | 2012-10-04 | Compositions and methods for silencing aldehyde dehydrogenase |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2014117690A true RU2014117690A (ru) | 2015-11-10 |
Family
ID=48044157
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2014117690/10A RU2014117690A (ru) | 2011-10-05 | 2012-10-04 | Композиции и способы для сайленсинга альдегид-дегидрогеназы |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US8999950B2 (https=) |
| EP (1) | EP2764007A4 (https=) |
| JP (1) | JP2014530602A (https=) |
| KR (1) | KR20140097143A (https=) |
| CN (1) | CN104114571A (https=) |
| AU (2) | AU2012318249B2 (https=) |
| CA (1) | CA2850792A1 (https=) |
| HK (1) | HK1200837A1 (https=) |
| RU (1) | RU2014117690A (https=) |
| WO (1) | WO2013052677A1 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2788030C2 (ru) * | 2015-12-13 | 2023-01-16 | Нитто Денко Корпорейшн | Структуры мирнк с высокой активностью и сниженным воздействием вне мишени |
| US11926831B2 (en) | 2015-12-13 | 2024-03-12 | Nitto Denko Corporation | SiRNA structures for high activity and reduced off target |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2009238175C1 (en) | 2008-04-15 | 2023-11-30 | Arbutus Biopharma Corporation | Novel lipid formulations for nucleic acid delivery |
| ES2613498T3 (es) | 2009-07-01 | 2017-05-24 | Protiva Biotherapeutics Inc. | Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos |
| HUE042177T2 (hu) | 2009-12-01 | 2019-06-28 | Translate Bio Inc | Szteroidszármazék mRNS szállítására humán genetikai betegségekben |
| WO2012000104A1 (en) | 2010-06-30 | 2012-01-05 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| PL3586861T3 (pl) | 2011-06-08 | 2022-05-23 | Translate Bio, Inc. | Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna |
| EP3354644A1 (en) | 2011-06-08 | 2018-08-01 | Translate Bio, Inc. | Cleavable lipids |
| ES2762873T3 (es) | 2012-03-29 | 2020-05-26 | Translate Bio Inc | Lípidos catiónicos ionizables |
| HK1206644A1 (en) | 2012-03-29 | 2016-01-15 | Shire Human Genetic Therapies, Inc. | Lipid-derived neutral nanoparticles |
| EP3536787A1 (en) | 2012-06-08 | 2019-09-11 | Translate Bio, Inc. | Nuclease resistant polynucleotides and uses thereof |
| US10245229B2 (en) | 2012-06-08 | 2019-04-02 | Translate Bio, Inc. | Pulmonary delivery of mRNA to non-lung target cells |
| PL3467108T3 (pl) | 2013-03-14 | 2024-09-30 | Translate Bio, Inc. | Sposoby oczyszczania informacyjnego rna |
| KR20210122917A (ko) | 2013-03-14 | 2021-10-12 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
| AU2014340149B2 (en) | 2013-10-22 | 2020-12-24 | Shire Human Genetic Therapies, Inc. | CNS delivery of mRNA and uses thereof |
| ES2707966T3 (es) | 2013-10-22 | 2019-04-08 | Translate Bio Inc | Terapia de ARNm para la deficiencia en síntesis de argininosuccinato |
| EA034103B1 (ru) | 2013-10-22 | 2019-12-27 | Транслейт Био, Инк. | СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК |
| MX2016005238A (es) | 2013-10-22 | 2016-08-12 | Shire Human Genetic Therapies | Formulaciones de lipidos para la administracion de acido ribonucleico mensajero. |
| PT3134506T (pt) | 2014-04-25 | 2019-10-31 | Translate Bio Inc | Métodos de purificação de rna mensageiro |
| JP6557722B2 (ja) | 2014-05-30 | 2019-08-07 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 核酸の送達のための生分解性脂質 |
| EP3160959B1 (en) | 2014-06-24 | 2023-08-30 | Translate Bio, Inc. | Stereochemically enriched compositions for delivery of nucleic acids |
| BR112016030852A2 (pt) | 2014-07-02 | 2018-01-16 | Shire Human Genetic Therapies | encapsulação de rna mensageiro |
| WO2016090262A1 (en) | 2014-12-05 | 2016-06-09 | Shire Human Genetic Therapies, Inc. | Messenger rna therapy for treatment of articular disease |
| US10172924B2 (en) | 2015-03-19 | 2019-01-08 | Translate Bio, Inc. | MRNA therapy for pompe disease |
| WO2016183366A2 (en) * | 2015-05-12 | 2016-11-17 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing expression of hepatitis d virus rna |
| CN114686548A (zh) | 2015-10-14 | 2022-07-01 | 川斯勒佰尔公司 | 用于增强生产的rna相关酶的修饰 |
| EA201991747A1 (ru) | 2017-02-27 | 2020-06-04 | Транслейт Био, Инк. | НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR |
| EP3483269A1 (en) * | 2017-11-13 | 2019-05-15 | Silence Therapeutics GmbH | Products and compositions |
| MX2019013752A (es) | 2017-05-16 | 2020-07-20 | Translate Bio Inc | Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones. |
| CA3118327A1 (en) * | 2017-11-13 | 2019-05-16 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of aldh2 in a cell |
| WO2019143621A1 (en) * | 2018-01-16 | 2019-07-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting aldh2 expression |
| AU2019227510B2 (en) * | 2018-03-02 | 2026-02-12 | Novo Nordisk A/S | Compositions and methods for inhibiting GYS2 expression |
| CA3108544A1 (en) | 2018-08-24 | 2020-02-27 | Translate Bio, Inc. | Methods for purification of messenger rna |
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| MX2021005969A (es) | 2018-11-21 | 2021-09-14 | Translate Bio Inc | Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado. |
| CA3135402A1 (en) * | 2019-04-04 | 2020-10-08 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression in the central nervous system |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2559828T3 (es) * | 2003-07-16 | 2016-02-16 | Protiva Biotherapeutics Inc. | ARN de interferencia encapsulado en lípidos |
| US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| US8101741B2 (en) * | 2005-11-02 | 2012-01-24 | Protiva Biotherapeutics, Inc. | Modified siRNA molecules and uses thereof |
| US20100015218A1 (en) * | 2007-02-16 | 2010-01-21 | Vasant Jadhav | Compositions and methods for potentiated activity of biologically active molecules |
| AU2009238175C1 (en) * | 2008-04-15 | 2023-11-30 | Arbutus Biopharma Corporation | Novel lipid formulations for nucleic acid delivery |
| US20110071208A1 (en) * | 2009-06-05 | 2011-03-24 | Protiva Biotherapeutics, Inc. | Lipid encapsulated dicer-substrate interfering rna |
| NZ597504A (en) * | 2009-06-15 | 2013-10-25 | Alnylam Pharmaceuticals Inc | Lipid formulated dsrna targeting the pcsk9 gene |
| US8236943B2 (en) * | 2009-07-01 | 2012-08-07 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing apolipoprotein B |
| WO2011011447A1 (en) * | 2009-07-20 | 2011-01-27 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing ebola virus gene expression |
-
2012
- 2012-10-04 US US14/349,619 patent/US8999950B2/en not_active Expired - Fee Related
- 2012-10-04 JP JP2014534724A patent/JP2014530602A/ja active Pending
- 2012-10-04 KR KR1020147011617A patent/KR20140097143A/ko not_active Withdrawn
- 2012-10-04 RU RU2014117690/10A patent/RU2014117690A/ru not_active Application Discontinuation
- 2012-10-04 CA CA2850792A patent/CA2850792A1/en not_active Abandoned
- 2012-10-04 EP EP12838796.6A patent/EP2764007A4/en not_active Withdrawn
- 2012-10-04 AU AU2012318249A patent/AU2012318249B2/en not_active Expired - Fee Related
- 2012-10-04 WO PCT/US2012/058770 patent/WO2013052677A1/en not_active Ceased
- 2012-10-04 HK HK15101352.3A patent/HK1200837A1/xx unknown
- 2012-10-04 CN CN201280059878.1A patent/CN104114571A/zh active Pending
-
2016
- 2016-03-29 AU AU2016201913A patent/AU2016201913A1/en not_active Abandoned
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2788030C2 (ru) * | 2015-12-13 | 2023-01-16 | Нитто Денко Корпорейшн | Структуры мирнк с высокой активностью и сниженным воздействием вне мишени |
| US11926831B2 (en) | 2015-12-13 | 2024-03-12 | Nitto Denko Corporation | SiRNA structures for high activity and reduced off target |
Also Published As
| Publication number | Publication date |
|---|---|
| AU2016201913A1 (en) | 2016-04-21 |
| US20140248338A1 (en) | 2014-09-04 |
| JP2014530602A (ja) | 2014-11-20 |
| NZ623181A (en) | 2015-11-27 |
| CA2850792A1 (en) | 2013-04-11 |
| AU2012318249B2 (en) | 2016-04-21 |
| KR20140097143A (ko) | 2014-08-06 |
| CN104114571A (zh) | 2014-10-22 |
| AU2012318249A1 (en) | 2013-05-02 |
| EP2764007A1 (en) | 2014-08-13 |
| HK1200837A1 (en) | 2015-08-14 |
| WO2013052677A1 (en) | 2013-04-11 |
| US8999950B2 (en) | 2015-04-07 |
| EP2764007A4 (en) | 2015-05-06 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20170111 |