AU2012318249B2 - Compositions and methods for silencing aldehyde dehydrogenase - Google Patents
Compositions and methods for silencing aldehyde dehydrogenase Download PDFInfo
- Publication number
- AU2012318249B2 AU2012318249B2 AU2012318249A AU2012318249A AU2012318249B2 AU 2012318249 B2 AU2012318249 B2 AU 2012318249B2 AU 2012318249 A AU2012318249 A AU 2012318249A AU 2012318249 A AU2012318249 A AU 2012318249A AU 2012318249 B2 AU2012318249 B2 AU 2012318249B2
- Authority
- AU
- Australia
- Prior art keywords
- lipid
- mol
- sirna
- nucleotides
- particle
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Expired - Fee Related
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)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Addiction (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (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)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2016201913A AU2016201913A1 (en) | 2011-10-05 | 2016-03-29 | Compositions and methods for silencing aldehyde dehydrogenase |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016201913A Division AU2016201913A1 (en) | 2011-10-05 | 2016-03-29 | Compositions and methods for silencing aldehyde dehydrogenase |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2012318249A1 AU2012318249A1 (en) | 2013-05-02 |
| AU2012318249B2 true AU2012318249B2 (en) | 2016-04-21 |
Family
ID=48044157
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2012318249A Expired - Fee Related AU2012318249B2 (en) | 2011-10-05 | 2012-10-04 | Compositions and methods for silencing aldehyde dehydrogenase |
| AU2016201913A Abandoned AU2016201913A1 (en) | 2011-10-05 | 2016-03-29 | Compositions and methods for silencing aldehyde dehydrogenase |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016201913A Abandoned AU2016201913A1 (en) | 2011-10-05 | 2016-03-29 | Compositions and methods for silencing aldehyde dehydrogenase |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US8999950B2 (enExample) |
| EP (1) | EP2764007A4 (enExample) |
| JP (1) | JP2014530602A (enExample) |
| KR (1) | KR20140097143A (enExample) |
| CN (1) | CN104114571A (enExample) |
| AU (2) | AU2012318249B2 (enExample) |
| CA (1) | CA2850792A1 (enExample) |
| HK (1) | HK1200837A1 (enExample) |
| RU (1) | RU2014117690A (enExample) |
| WO (1) | WO2013052677A1 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2279254T3 (pl) | 2008-04-15 | 2017-11-30 | Protiva Biotherapeutics Inc. | Nowe preparaty lipidowe do dostarczania kwasów nukleinowych |
| EP2449114B9 (en) | 2009-07-01 | 2017-04-19 | Protiva Biotherapeutics Inc. | Novel lipid formulations for delivery of therapeutic agents to solid tumors |
| NZ700688A (en) | 2009-12-01 | 2016-02-26 | Shire Human Genetic Therapies | 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 |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| EP3336082B1 (en) | 2011-06-08 | 2020-04-15 | Translate Bio, Inc. | Cleavable lipids |
| CN103906527B (zh) | 2011-06-08 | 2020-07-10 | 川斯勒佰尔公司 | Mrna递送的脂质纳米颗粒组合物和方法 |
| JP6211054B2 (ja) | 2012-03-29 | 2017-10-11 | 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 | 脂質誘導される中性ナノ粒子 |
| BR112014024131A2 (pt) | 2012-03-29 | 2017-07-25 | Shire Human Genetic Therapies | lipídios catiônicos ionizáveis |
| EP3884949A1 (en) | 2012-06-08 | 2021-09-29 | Translate Bio, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| US20150267192A1 (en) | 2012-06-08 | 2015-09-24 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| ES2708561T3 (es) | 2013-03-14 | 2019-04-10 | Translate Bio Inc | Métodos para la purificación de ARN mensajero |
| EP2968586B1 (en) | 2013-03-14 | 2018-07-25 | Translate Bio, Inc. | Cftr mrna compositions and related methods and uses |
| EP3060671B1 (en) | 2013-10-22 | 2021-12-29 | Translate Bio, Inc. | Cns delivery of mrna and uses thereof |
| EP3060258A1 (en) | 2013-10-22 | 2016-08-31 | Shire Human Genetic Therapies, Inc. | Mrna therapy for phenylketonuria |
| EP4276176A3 (en) | 2013-10-22 | 2024-01-10 | Translate Bio, Inc. | Mrna therapy for argininosuccinate synthetase deficiency |
| KR101988552B1 (ko) | 2013-10-22 | 2019-09-25 | 샤이어 휴먼 지네틱 테라피즈 인크. | 메신저 rna의 전달을 위한 지질 제형 |
| CA2944800A1 (en) | 2014-04-25 | 2015-10-29 | Shire Human Genetic Therapies, Inc. | Methods for purification of messenger rna |
| EP3148552B1 (en) | 2014-05-30 | 2019-07-31 | Translate Bio, Inc. | Biodegradable lipids for delivery of nucleic acids |
| KR102511554B1 (ko) | 2014-06-24 | 2023-03-16 | 샤이어 휴먼 지네틱 테라피즈 인크. | 핵산의 전달용 입체화학적으로 풍부한 조성물 |
| CA2953265C (en) | 2014-07-02 | 2023-09-26 | Shire Human Genetic Therapies, Inc. | Encapsulation of messenger rna |
| US9943595B2 (en) | 2014-12-05 | 2018-04-17 | Translate Bio, Inc. | Messenger RNA therapy for treatment of articular disease |
| JP6895892B2 (ja) | 2015-03-19 | 2021-06-30 | トランスレイト バイオ, インコーポレイテッド | ポンペ病のmRNA治療 |
| WO2016183366A2 (en) * | 2015-05-12 | 2016-11-17 | Protiva Biotherapeutics, Inc. | Compositions and methods for silencing expression of hepatitis d virus rna |
| AU2016338559B2 (en) | 2015-10-14 | 2022-11-24 | Translate Bio, Inc. | Modification of RNA-related enzymes for enhanced production |
| PT3386519T (pt) | 2015-12-13 | 2021-04-27 | Nitto Denko Corp | Estruturas de sirna para atividade elevada e redução de alvos extra |
| 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 |
| WO2018213476A1 (en) | 2017-05-16 | 2018-11-22 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr |
| WO2019092282A1 (en) * | 2017-11-13 | 2019-05-16 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of aldh2 in a cell |
| EP3710588A4 (en) * | 2018-01-16 | 2021-08-18 | Dicerna Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS TO INHIBIT ALDH2 EXPRESSION |
| US11572562B2 (en) * | 2018-03-02 | 2023-02-07 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting GYS2 expression |
| WO2020041793A1 (en) | 2018-08-24 | 2020-02-27 | Translate Bio, Inc. | Methods for purification of messenger rna |
| EP3864163B1 (en) | 2018-10-09 | 2024-03-20 | The University of British Columbia | Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto |
| EP3883592A1 (en) | 2018-11-21 | 2021-09-29 | 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 |
|---|---|---|---|---|
| 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 |
| AU2006308765B2 (en) * | 2005-11-02 | 2013-09-05 | Arbutus Biopharma Corporation | Modified siRNA molecules and uses thereof |
| EP2131848A4 (en) * | 2007-02-16 | 2012-06-27 | Merck Sharp & Dohme | COMPOSITIONS AND METHODS FOR REINFORCED ACTIVITY OF BIOLOGICAL ACTIVE MOLECULES |
| PL2279254T3 (pl) * | 2008-04-15 | 2017-11-30 | Protiva Biotherapeutics Inc. | Nowe preparaty lipidowe do dostarczania kwasów nukleinowych |
| US20110071208A1 (en) * | 2009-06-05 | 2011-03-24 | Protiva Biotherapeutics, Inc. | Lipid encapsulated dicer-substrate interfering rna |
| CN104651408A (zh) * | 2009-06-15 | 2015-05-27 | 阿尔尼拉姆医药品有限公司 | 靶向pcsk9基因的脂质配制的dsrna |
| 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 CN CN201280059878.1A patent/CN104114571A/zh active Pending
- 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 KR KR1020147011617A patent/KR20140097143A/ko not_active Withdrawn
- 2012-10-04 HK HK15101352.3A patent/HK1200837A1/xx unknown
- 2012-10-04 WO PCT/US2012/058770 patent/WO2013052677A1/en not_active Ceased
- 2012-10-04 EP EP12838796.6A patent/EP2764007A4/en not_active Withdrawn
- 2012-10-04 CA CA2850792A patent/CA2850792A1/en not_active Abandoned
- 2012-10-04 US US14/349,619 patent/US8999950B2/en not_active Expired - Fee Related
- 2012-10-04 AU AU2012318249A patent/AU2012318249B2/en not_active Expired - Fee Related
-
2016
- 2016-03-29 AU AU2016201913A patent/AU2016201913A1/en not_active Abandoned
Non-Patent Citations (1)
| Title |
|---|
| Cortinez, G. et al., Alcohol, 2009, Vol. 43, pages 97-104 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2013052677A1 (en) | 2013-04-11 |
| NZ623181A (en) | 2015-11-27 |
| HK1200837A1 (en) | 2015-08-14 |
| KR20140097143A (ko) | 2014-08-06 |
| RU2014117690A (ru) | 2015-11-10 |
| US8999950B2 (en) | 2015-04-07 |
| JP2014530602A (ja) | 2014-11-20 |
| CA2850792A1 (en) | 2013-04-11 |
| EP2764007A4 (en) | 2015-05-06 |
| AU2012318249A1 (en) | 2013-05-02 |
| CN104114571A (zh) | 2014-10-22 |
| AU2016201913A1 (en) | 2016-04-21 |
| EP2764007A1 (en) | 2014-08-13 |
| US20140248338A1 (en) | 2014-09-04 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MK25 | Application lapsed reg. 22.2i(2) - failure to pay acceptance fee |