HK1200837A1 - Compositions and methods for silencing aldehyde dehydrogenase - Google Patents
Compositions and methods for silencing aldehyde dehydrogenase Download PDFInfo
- Publication number
- HK1200837A1 HK1200837A1 HK15101352.3A HK15101352A HK1200837A1 HK 1200837 A1 HK1200837 A1 HK 1200837A1 HK 15101352 A HK15101352 A HK 15101352A HK 1200837 A1 HK1200837 A1 HK 1200837A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- methods
- aldehyde dehydrogenase
- compositions
- silencing
- lipid particles
- 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)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Addiction (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161543700P | 2011-10-05 | 2011-10-05 | |
| US201161543700P | 2011-10-05 | ||
| US201261599238P | 2012-02-15 | 2012-02-15 | |
| US201261599238P | 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 |
|---|---|
| HK1200837A1 true HK1200837A1 (en) | 2015-08-14 |
Family
ID=48044157
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HK15101352.3A HK1200837A1 (en) | 2011-10-05 | 2012-10-04 | Compositions and methods for silencing aldehyde dehydrogenase |
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=) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | Protiva Biotherapeutics, Inc. | Novel lipid formulations for nucleic acid delivery |
| WO2011000107A1 (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 |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| ES2795110T3 (es) | 2011-06-08 | 2020-11-20 | Translate Bio Inc | Lípidos escindibles |
| BR112014024131A2 (pt) | 2012-03-29 | 2017-07-25 | Shire Human Genetic Therapies | lipídios catiônicos ionizáveis |
| AU2013237874B2 (en) | 2012-03-29 | 2018-01-18 | Translate Bio, Inc. | Lipid-derived neutral nanoparticles |
| WO2013185067A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| EP2858679B2 (en) | 2012-06-08 | 2024-06-05 | Translate Bio, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| JP6586075B2 (ja) | 2013-03-14 | 2019-10-02 | 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 |
| RS57739B1 (sr) | 2013-03-14 | 2018-12-31 | Translate Bio Inc | Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima |
| JP6506749B2 (ja) | 2013-10-22 | 2019-04-24 | 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 | フェニルケトン尿症のためのｍＲＮＡ療法 |
| CN106413811A (zh) | 2013-10-22 | 2017-02-15 | 夏尔人类遗传性治疗公司 | 精氨基琥珀酸合成酶缺乏症的mrna疗法 |
| AU2014340149B2 (en) | 2013-10-22 | 2020-12-24 | Shire Human Genetic Therapies, Inc. | CNS delivery of mRNA and uses thereof |
| ES3032935T3 (en) | 2013-10-22 | 2025-07-29 | Translate Bio Inc | Lipid formulations for delivery of messenger rna |
| US9850269B2 (en) | 2014-04-25 | 2017-12-26 | Translate Bio, Inc. | Methods for purification of messenger RNA |
| 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 |
| KR102559979B1 (ko) | 2014-06-24 | 2023-07-25 | 샤이어 휴먼 지네틱 테라피즈 인크. | 핵산의 전달용 입체화학적으로 풍부한 조성물 |
| BR112016030852A2 (pt) | 2014-07-02 | 2018-01-16 | Shire Human Genetic Therapies | encapsulação de rna mensageiro |
| JP6767976B2 (ja) | 2014-12-05 | 2020-10-14 | トランスレイト バイオ， インコーポレイテッド | 関節疾患の治療のためのメッセンジャーｒｎａ治療法 |
| 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 |
| JP6997704B2 (ja) | 2015-10-14 | 2022-02-04 | トランスレイト バイオ， インコーポレイテッド | 生産性向上のためのｒｎａ関連酵素の修飾 |
| BR112018008344A2 (pt) | 2015-12-13 | 2018-10-30 | Nitto Denko Corp | molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição |
| 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. |
| EP3735461A1 (en) * | 2017-11-13 | 2020-11-11 | Silence Therapeutics GmbH | Nucleic acids for inhibiting expression of aldh2 in a cell |
| KR20200109311A (ko) * | 2018-01-16 | 2020-09-22 | 다이서나 파마수이티컬, 인크. | Aldh2 발현을 억제하기 위한 조성물 및 방법 |
| CA3092092A1 (en) * | 2018-03-02 | 2019-09-06 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting gys2 expression |
| KR102950141B1 (ko) | 2018-08-24 | 2026-04-07 | 트랜슬레이트 바이오 인코포레이티드 | 전령 rna의 정제 방법 |
| HRP20240586T1 (hr) | 2018-10-09 | 2024-07-19 | The University Of British Columbia | Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima |
| WO2020106946A1 (en) | 2018-11-21 | 2020-05-28 | Translate Bio, Inc. | TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR |
| BR112021019793A2 (pt) * | 2019-04-04 | 2021-12-07 | Dicerna Pharmaceuticals Inc | Composições e métodos para inibir expressão de gene no sistema nervoso central |
| EP4031662A1 (en) | 2019-09-20 | 2022-07-27 | Translate Bio, Inc. | Mrna encoding engineered cftr |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2532228C (en) * | 2003-07-16 | 2017-02-14 | 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 |
| CN101346393B (zh) * | 2005-11-02 | 2015-07-22 | 普洛体维生物治疗公司 | 修饰的siRNA分子及其应用 |
| CA2689042A1 (en) * | 2007-02-16 | 2008-08-28 | Merck & Co., Inc. | Compositions and methods for potentiated activity of biologicaly active molecules |
| CA2721333C (en) * | 2008-04-15 | 2020-12-01 | 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 |
| JP5894913B2 (ja) * | 2009-06-15 | 2016-03-30 | アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | Ｐｃｓｋ９遺伝子を標的とする、脂質で製剤化されたｄｓｒｎａ |
| EP2449106B1 (en) * | 2009-07-01 | 2015-04-08 | Protiva Biotherapeutics Inc. | Compositions and methods for silencing apolipoprotein b |
| US8716464B2 (en) | 2009-07-20 | 2014-05-06 | Thomas W. Geisbert | Compositions and methods for silencing Ebola virus gene expression |
-
2012
- 2012-10-04 JP JP2014534724A patent/JP2014530602A/ja active Pending
- 2012-10-04 KR KR1020147011617A patent/KR20140097143A/ko not_active Withdrawn
- 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 CN CN201280059878.1A patent/CN104114571A/zh active Pending
- 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 US US14/349,619 patent/US8999950B2/en not_active Expired - Fee Related
- 2012-10-04 RU RU2014117690/10A patent/RU2014117690A/ru not_active Application Discontinuation
- 2012-10-04 HK HK15101352.3A patent/HK1200837A1/xx unknown
-
2016
- 2016-03-29 AU AU2016201913A patent/AU2016201913A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| KR20140097143A (ko) | 2014-08-06 |
| RU2014117690A (ru) | 2015-11-10 |
| AU2012318249B2 (en) | 2016-04-21 |
| AU2016201913A1 (en) | 2016-04-21 |
| CN104114571A (zh) | 2014-10-22 |
| JP2014530602A (ja) | 2014-11-20 |
| EP2764007A1 (en) | 2014-08-13 |
| US20140248338A1 (en) | 2014-09-04 |
| EP2764007A4 (en) | 2015-05-06 |
| NZ623181A (en) | 2015-11-27 |
| WO2013052677A1 (en) | 2013-04-11 |
| CA2850792A1 (en) | 2013-04-11 |
| US8999950B2 (en) | 2015-04-07 |
| AU2012318249A1 (en) | 2013-05-02 |
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