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)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Addiction (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 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2764007A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2014530602A (cg-RX-API-DMAC7.html) |
| KR (1) | KR20140097143A (cg-RX-API-DMAC7.html) |
| CN (1) | CN104114571A (cg-RX-API-DMAC7.html) |
| AU (2) | AU2012318249B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2850792A1 (cg-RX-API-DMAC7.html) |
| HK (1) | HK1200837A1 (cg-RX-API-DMAC7.html) |
| RU (1) | RU2014117690A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2013052677A1 (cg-RX-API-DMAC7.html) |
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 |
|---|---|---|---|---|
| JP5475753B2 (ja) | 2008-04-15 | 2014-04-16 | プロチバ バイオセラピューティクス インコーポレイティッド | 核酸送達用の脂質製剤 |
| CA2767127A1 (en) | 2009-07-01 | 2011-01-06 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for delivery of therapeutic agents to solid tumors |
| EP3318248B1 (en) | 2009-12-01 | 2019-04-10 | Translate Bio, Inc. | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| US9006417B2 (en) | 2010-06-30 | 2015-04-14 | 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 |
| CA3198966A1 (en) | 2011-06-08 | 2012-12-13 | Translate Bio, Inc. | Cleavable lipids |
| EP2717893B1 (en) | 2011-06-08 | 2019-05-08 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| CA2868034C (en) | 2012-03-29 | 2021-07-27 | Shire Human Genetic Therapies, Inc. | Ionizable cationic lipids |
| HK1206644A1 (en) | 2012-03-29 | 2016-01-15 | Shire Human Genetic Therapies, Inc. | Lipid-derived neutral nanoparticles |
| WO2013185067A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| WO2013185069A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| TR201901310T4 (tr) | 2013-03-14 | 2019-02-21 | Translate Bio Inc | Mesajcı RNA'nın saflaştırılması yöntemleri. |
| LT2968586T (lt) | 2013-03-14 | 2018-11-26 | Translate Bio, Inc. | Cft mrnr kompozicijos bei su jomis susiję būdai ir panaudojimai |
| BR112016009014B1 (pt) | 2013-10-22 | 2024-02-06 | Translate Bio, Inc | USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE |
| CN112618732A (zh) | 2013-10-22 | 2021-04-09 | 夏尔人类遗传性治疗公司 | 用于递送信使rna的脂质制剂 |
| EP3574923A1 (en) | 2013-10-22 | 2019-12-04 | Translate Bio, Inc. | Mrna therapy for phenylketonuria |
| CN105658800A (zh) | 2013-10-22 | 2016-06-08 | 夏尔人类遗传性治疗公司 | Mrna的cns递送及其用途 |
| KR102739894B1 (ko) | 2014-04-25 | 2024-12-05 | 샤이어 휴먼 지네틱 테라피즈 인크. | 메신저 rna 의 정제 방법 |
| US10022455B2 (en) | 2014-05-30 | 2018-07-17 | Translate Bio, Inc. | Biodegradable lipids for delivery of nucleic acids |
| CN111588695A (zh) | 2014-06-24 | 2020-08-28 | 川斯勒佰尔公司 | 用于递送核酸的立体化学富集组合物 |
| 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 |
| AU2016233135B2 (en) | 2015-03-19 | 2021-07-08 | 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 |
| CA3001852A1 (en) | 2015-10-14 | 2017-04-20 | Translate Bio, Inc. | Modification of rna-related enzymes for enhanced production |
| EP3585417B1 (en) | 2017-02-27 | 2023-02-22 | Translate Bio, Inc. | Method of making a codon-optimized cftr mrna |
| EP3483269A1 (en) * | 2017-11-13 | 2019-05-15 | Silence Therapeutics GmbH | Products and compositions |
| CA3063531A1 (en) | 2017-05-16 | 2018-11-22 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr |
| EP3735461A1 (en) * | 2017-11-13 | 2020-11-11 | Silence Therapeutics GmbH | Nucleic acids for inhibiting expression of aldh2 in a cell |
| AU2019209324A1 (en) * | 2018-01-16 | 2020-07-09 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting ALDH2 expression |
| MX2020009072A (es) * | 2018-03-02 | 2020-10-08 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de gys2. |
| CA3108544A1 (en) | 2018-08-24 | 2020-02-27 | Translate Bio, Inc. | Methods for purification of messenger rna |
| EP4379053A3 (en) | 2018-10-09 | 2024-09-18 | The University of British Columbia | Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto |
| CA3120647A1 (en) | 2018-11-21 | 2020-05-28 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr |
| US20220170025A1 (en) * | 2019-04-04 | 2022-06-02 | 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 |
|---|---|---|---|---|
| JP4951338B2 (ja) * | 2003-07-16 | 2012-06-13 | プロチバ バイオセラピューティクス インコーポレイティッド | 脂質に封入された干渉ｒｎａ |
| US7404969B2 (en) * | 2005-02-14 | 2008-07-29 | Sirna Therapeutics, Inc. | Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules |
| EP1948674A4 (en) * | 2005-11-02 | 2009-02-04 | Protiva Biotherapeutics Inc | MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF |
| AU2008219165A1 (en) * | 2007-02-16 | 2008-08-28 | Merck Sharp & Dohme Corp. | Compositions and methods for potentiated activity of biologicaly active molecules |
| JP5475753B2 (ja) * | 2008-04-15 | 2014-04-16 | プロチバ バイオセラピューティクス インコーポレイティッド | 核酸送達用の脂質製剤 |
| 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 |
| EP2449106B1 (en) * | 2009-07-01 | 2015-04-08 | 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 AU AU2012318249A patent/AU2012318249B2/en not_active Expired - Fee Related
- 2012-10-04 CN CN201280059878.1A patent/CN104114571A/zh active Pending
- 2012-10-04 US US14/349,619 patent/US8999950B2/en not_active Expired - Fee Related
- 2012-10-04 EP EP12838796.6A patent/EP2764007A4/en not_active Withdrawn
- 2012-10-04 WO PCT/US2012/058770 patent/WO2013052677A1/en not_active Ceased
- 2012-10-04 KR KR1020147011617A patent/KR20140097143A/ko not_active Withdrawn
- 2012-10-04 JP JP2014534724A patent/JP2014530602A/ja active Pending
- 2012-10-04 RU RU2014117690/10A patent/RU2014117690A/ru not_active Application Discontinuation
- 2012-10-04 HK HK15101352.3A patent/HK1200837A1/xx unknown
- 2012-10-04 CA CA2850792A patent/CA2850792A1/en not_active Abandoned
-
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 |
|---|---|
| NZ623181A (en) | 2015-11-27 |
| CN104114571A (zh) | 2014-10-22 |
| EP2764007A4 (en) | 2015-05-06 |
| KR20140097143A (ko) | 2014-08-06 |
| AU2012318249A1 (en) | 2013-05-02 |
| CA2850792A1 (en) | 2013-04-11 |
| AU2012318249B2 (en) | 2016-04-21 |
| US8999950B2 (en) | 2015-04-07 |
| WO2013052677A1 (en) | 2013-04-11 |
| HK1200837A1 (en) | 2015-08-14 |
| JP2014530602A (ja) | 2014-11-20 |
| AU2016201913A1 (en) | 2016-04-21 |
| US20140248338A1 (en) | 2014-09-04 |
| EP2764007A1 (en) | 2014-08-13 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA92 | Acknowledgement of application withdrawn (lack of supplementary materials submitted) |
Effective date: 20170111 |