CN104114571A - 用于沉默醛脱氢酶的组合物和方法 - Google Patents

用于沉默醛脱氢酶的组合物和方法 Download PDF

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Publication number
CN104114571A
CN104114571A CN201280059878.1A CN201280059878A CN104114571A CN 104114571 A CN104114571 A CN 104114571A CN 201280059878 A CN201280059878 A CN 201280059878A CN 104114571 A CN104114571 A CN 104114571A
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lipid
nucleic acid
particle
mole
nucleotides
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Pending
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CN201280059878.1A
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English (en)
Chinese (zh)
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伊恩·麦克拉克伦
埃米·C·H·李
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Protiva Biotherapeutics Inc
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Protiva Biotherapeutics Inc
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    • 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)

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  • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Addiction (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Neurology (AREA)
  • Psychiatry (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN201280059878.1A 2011-10-05 2012-10-04 用于沉默醛脱氢酶的组合物和方法 Pending CN104114571A (zh)

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
CN104114571A true CN104114571A (zh) 2014-10-22

Family

ID=48044157

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280059878.1A Pending CN104114571A (zh) 2011-10-05 2012-10-04 用于沉默醛脱氢酶的组合物和方法

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)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111601891A (zh) * 2018-01-16 2020-08-28 迪克纳制药公司 用于抑制aldh2表达的组合物和方法
CN113748208A (zh) * 2019-04-04 2021-12-03 迪克纳制药公司 用于抑制中枢神经系统中的基因表达的组合物和方法

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. 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
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment 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
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
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
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
KR102311614B1 (ko) 2013-03-14 2021-10-08 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
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EP4729528A3 (en) 2014-04-25 2026-06-24 Translate Bio, Inc. Methods for purification of messenger rna
WO2015184256A2 (en) 2014-05-30 2015-12-03 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
WO2016004318A1 (en) 2014-07-02 2016-01-07 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
AU2015357562B2 (en) 2014-12-05 2021-10-21 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
EP3270894B1 (en) 2015-03-19 2021-02-24 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
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
AU2016371624B2 (en) 2015-12-13 2020-08-27 Nitto Denko Corporation siRNA structures for high activity and reduced off target
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
EP3483269A1 (en) * 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
IL270631B2 (en) 2017-05-16 2024-03-01 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
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
US12678406B2 (en) 2020-10-12 2026-07-14 Translate Bio Inc. Process of preparing ice-based lipid nanoparticles

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101346393A (zh) * 2005-11-02 2009-01-14 普洛体维生物治疗公司 修饰的siRNA分子及其应用
CN102119217A (zh) * 2008-04-15 2011-07-06 普洛体维生物治疗公司 用于核酸递送的新型制剂
US20110201667A1 (en) * 2009-07-20 2011-08-18 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004257373B2 (en) * 2003-07-16 2011-03-24 Arbutus Biopharma Corporation 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
WO2008103276A2 (en) * 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101346393A (zh) * 2005-11-02 2009-01-14 普洛体维生物治疗公司 修饰的siRNA分子及其应用
CN102119217A (zh) * 2008-04-15 2011-07-06 普洛体维生物治疗公司 用于核酸递送的新型制剂
US20110201667A1 (en) * 2009-07-20 2011-08-18 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
GABRIEL CORTINEZ等: "RNA interference against aldehyde dehydrogenase-2: development of tools for alcohol research", 《ALCOHOL》 *
GABRIEL CORTINEZ等: "RNA interference against aldehyde dehydrogenase-2: development of tools for alcohol research", 《ALCOHOL》, vol. 43, 31 December 2009 (2009-12-31), pages 97 - 104, XP025992308, DOI: doi:10.1016/j.alcohol.2008.12.007 *
LILY C. HSU等: "Genomic Structure of the Human Mitochondrial Aldehyde Dehydrogenase Gene", 《GENOMICS》 *
毕煌垒等: "小分子干扰RNA分子化学修饰研究进展", 《国际药学研究杂志》 *
陈旭等: "不对称RNA干扰研究进展", 《药学与临床研究》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111601891A (zh) * 2018-01-16 2020-08-28 迪克纳制药公司 用于抑制aldh2表达的组合物和方法
CN113748208A (zh) * 2019-04-04 2021-12-03 迪克纳制药公司 用于抑制中枢神经系统中的基因表达的组合物和方法

Also Published As

Publication number Publication date
EP2764007A4 (en) 2015-05-06
US20140248338A1 (en) 2014-09-04
EP2764007A1 (en) 2014-08-13
KR20140097143A (ko) 2014-08-06
AU2012318249A1 (en) 2013-05-02
US8999950B2 (en) 2015-04-07
CA2850792A1 (en) 2013-04-11
JP2014530602A (ja) 2014-11-20
RU2014117690A (ru) 2015-11-10
AU2012318249B2 (en) 2016-04-21
NZ623181A (en) 2015-11-27
WO2013052677A1 (en) 2013-04-11
AU2016201913A1 (en) 2016-04-21
HK1200837A1 (en) 2015-08-14

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Application publication date: 20141022