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)
Inventor
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)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Addiction (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dispersion Chemistry (AREA)
  • Psychiatry (AREA)
  • Neurosurgery (AREA)
  • Neurology (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)

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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

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JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
CA2782676C (en) 2009-12-01 2021-06-15 Shire Human Genetic Therapies, Inc. Pharmaceutical compositions for intracellular delivery of nucleic acids and proteins and uses thereof for treatment of ureca cycle metabolic disorders
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
CA3107288A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
CN103748078B (zh) 2011-06-08 2016-11-09 夏尔人类遗传性治疗公司 可裂解脂质
US9546128B2 (en) 2012-03-29 2017-01-17 Shire Human Genetic Therapies, Inc. Ionizable cationic lipids
CA2868030C (en) 2012-03-29 2021-05-25 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
JP6561378B2 (ja) 2012-06-08 2019-08-21 トランスレイト バイオ， インコーポレイテッド 非肺標的細胞へのｍＲＮＡの経肺送達
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
MX365409B (es) 2013-03-14 2019-05-31 Shire Human Genetic Therapies Composiciones de ácido ribonucleico mensajero del regulador transmembrana de fibrosis quística y métodos y usos relacionados.
EP3871696B1 (en) 2013-10-22 2025-03-19 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
EP3060303B1 (en) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
KR20250005450A (ko) 2014-04-25 2025-01-09 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
BR112016027705A2 (pt) 2014-05-30 2018-01-30 Shire Human Genetic Therapies lipídios biodegradáveis para distribuição de ácidos nucleicos
PE20171238A1 (es) 2014-06-24 2017-08-24 Shire Human Genetic Therapies Composiciones enriquecidas estereoquimicamente para administracion de acidos nucleicos
CN114146063A (zh) 2014-07-02 2022-03-08 川斯勒佰尔公司 信使rna的包封
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, 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
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
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
CA3063531A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
CA3118327A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
CN112055598A (zh) * 2018-03-02 2020-12-08 迪克纳制药公司 用于抑制gys2表达的组合物和方法
EP3841208A1 (en) 2018-08-24 2021-06-30 Translate Bio, Inc. Methods for purification of messenger rna
EA202190688A1 (ru) 2018-10-09 2021-06-07 Дзе Юниверсити Оф Бритиш Коламбиа Композиции и системы, содержащие трансфекционно-компетентные везикулы, не содержащие органических растворителей и детергентов, и связанные с ними способы
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.

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

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Application publication date: 20141022