CA2850792A1 - Compositions and methods for silencing aldehyde dehydrogenase - Google Patents
Compositions and methods for silencing aldehyde dehydrogenase Download PDFInfo
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- CA2850792A1 CA2850792A1 CA2850792A CA2850792A CA2850792A1 CA 2850792 A1 CA2850792 A1 CA 2850792A1 CA 2850792 A CA2850792 A CA 2850792A CA 2850792 A CA2850792 A CA 2850792A CA 2850792 A1 CA2850792 A1 CA 2850792A1
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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
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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
- C12N15/113—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
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- 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
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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
- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- 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
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- Chemical Kinetics & Catalysis (AREA)
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- 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)
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| 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)
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| CA2850792A1 true CA2850792A1 (en) | 2013-04-11 |
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| CA2850792A Abandoned CA2850792A1 (en) | 2011-10-05 | 2012-10-04 | Compositions and methods for silencing aldehyde dehydrogenase |
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| EP (1) | EP2764007A4 (enExample) |
| JP (1) | JP2014530602A (enExample) |
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| HK (1) | HK1200837A1 (enExample) |
| RU (1) | RU2014117690A (enExample) |
| WO (1) | WO2013052677A1 (enExample) |
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| NZ588583A (en) | 2008-04-15 | 2012-08-31 | Protiva Biotherapeutics Inc | Novel lipid formulations for nucleic acid delivery |
| US8283333B2 (en) | 2009-07-01 | 2012-10-09 | Protiva Biotherapeutics, Inc. | Lipid formulations for nucleic acid delivery |
| PL3338765T3 (pl) | 2009-12-01 | 2019-06-28 | Translate Bio, Inc. | Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych |
| WO2012000104A1 (en) | 2010-06-30 | 2012-01-05 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| DK3336082T3 (da) | 2011-06-08 | 2020-04-27 | Translate Bio Inc | Spaltelige lipider |
| PT2717893T (pt) | 2011-06-08 | 2019-08-20 | Translate Bio Inc | Composições de nanopartículas lipídicas e métodos para transferência de arnm |
| ES2762873T3 (es) | 2012-03-29 | 2020-05-26 | Translate Bio Inc | Lípidos catiónicos ionizables |
| EP3865123A1 (en) | 2012-03-29 | 2021-08-18 | Translate Bio, Inc. | Lipid-derived neutral nanoparticles |
| WO2013185069A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| WO2013185067A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| EP2968586B1 (en) | 2013-03-14 | 2018-07-25 | Translate Bio, Inc. | Cftr mrna compositions and related methods and uses |
| ES2981185T3 (es) | 2013-03-14 | 2024-10-07 | Translate Bio Inc | Métodos para la purificación de ARN mensajero |
| CA2928040A1 (en) | 2013-10-22 | 2015-04-30 | Shire Human Genetic Therapies, Inc. | Cns delivery of mrna and uses thereof |
| US11224642B2 (en) | 2013-10-22 | 2022-01-18 | Translate Bio, Inc. | MRNA therapy for argininosuccinate synthetase deficiency |
| WO2015061467A1 (en) | 2013-10-22 | 2015-04-30 | Shire Human Genetic Therapies, Inc. | Lipid formulations for delivery of messenger rna |
| MX2016005239A (es) | 2013-10-22 | 2016-08-12 | Shire Human Genetic Therapies | Tratamiento con acido ribonucleico mensajero para la fenilcetonuria. |
| JP6571679B2 (ja) | 2014-04-25 | 2019-09-04 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａの精製方法 |
| US10022455B2 (en) | 2014-05-30 | 2018-07-17 | Translate Bio, Inc. | Biodegradable lipids for delivery of nucleic acids |
| CA2952824C (en) | 2014-06-24 | 2023-02-21 | Shire Human Genetic Therapies, Inc. | Stereochemically enriched compositions for delivery of nucleic acids |
| CA2953265C (en) | 2014-07-02 | 2023-09-26 | Shire Human Genetic Therapies, Inc. | Encapsulation of messenger rna |
| MA56412A (fr) | 2014-12-05 | 2022-05-04 | Translate Bio Inc | Thérapie par l'arn messager pour le traitement des maladies articulaires |
| WO2016149508A1 (en) | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, 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 |
| CN108473969B9 (zh) | 2015-10-14 | 2024-08-23 | 川斯勒佰尔公司 | 用于增强生产的rna相关酶的修饰 |
| US10358647B2 (en) | 2015-12-13 | 2019-07-23 | Nitto Denko Corporation | siRNA structures for high activity and reduced off target |
| AU2018224326B2 (en) | 2017-02-27 | 2024-01-04 | Translate Bio, Inc. | Novel codon-optimized CFTR mRNA |
| 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. |
| EP3735461A1 (en) * | 2017-11-13 | 2020-11-11 | 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 |
| CN118421617A (zh) | 2018-08-24 | 2024-08-02 | 川斯勒佰尔公司 | 用于纯化信使rna的方法 |
| 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 |
| EP3883592A1 (en) | 2018-11-21 | 2021-09-29 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr |
| WO2020206350A1 (en) * | 2019-04-04 | 2020-10-08 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression in the central nervous system |
| WO2021055609A1 (en) | 2019-09-20 | 2021-03-25 | Translate Bio, Inc. | Mrna encoding engineered cftr |
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| SG190613A1 (en) * | 2003-07-16 | 2013-06-28 | Protiva Biotherapeutics Inc | Lipid encapsulated interfering 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 |
| WO2007051303A1 (en) * | 2005-11-02 | 2007-05-10 | Protiva Biotherapeutics, Inc. | MODIFIED siRNA MOLECULES AND USES THEREOF |
| JP2010519203A (ja) * | 2007-02-16 | 2010-06-03 | メルク・シャープ・エンド・ドーム・コーポレイション | 生物活性分子の活性を強化するための組成物及び方法 |
| NZ588583A (en) * | 2008-04-15 | 2012-08-31 | Protiva Biotherapeutics Inc | Novel lipid formulations for nucleic acid delivery |
| US20110071208A1 (en) * | 2009-06-05 | 2011-03-24 | Protiva Biotherapeutics, Inc. | Lipid encapsulated dicer-substrate interfering rna |
| AU2010260148A1 (en) * | 2009-06-15 | 2012-02-02 | 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 |
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2012
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- 2012-10-04 WO PCT/US2012/058770 patent/WO2013052677A1/en not_active Ceased
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- 2012-10-04 HK HK15101352.3A patent/HK1200837A1/xx unknown
- 2012-10-04 CA CA2850792A patent/CA2850792A1/en not_active Abandoned
- 2012-10-04 EP EP12838796.6A patent/EP2764007A4/en not_active Withdrawn
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2016
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| RU2014117690A (ru) | 2015-11-10 |
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| AU2012318249B2 (en) | 2016-04-21 |
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| WO2013052677A1 (en) | 2013-04-11 |
| US8999950B2 (en) | 2015-04-07 |
| US20140248338A1 (en) | 2014-09-04 |
| CN104114571A (zh) | 2014-10-22 |
| JP2014530602A (ja) | 2014-11-20 |
| EP2764007A1 (en) | 2014-08-13 |
| KR20140097143A (ko) | 2014-08-06 |
| EP2764007A4 (en) | 2015-05-06 |
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