RU2014117690A - Композиции и способы для сайленсинга альдегид-дегидрогеназы - Google Patents

Композиции и способы для сайленсинга альдегид-дегидрогеназы Download PDF

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Publication number
RU2014117690A
RU2014117690A RU2014117690/10A RU2014117690A RU2014117690A RU 2014117690 A RU2014117690 A RU 2014117690A RU 2014117690/10 A RU2014117690/10 A RU 2014117690/10A RU 2014117690 A RU2014117690 A RU 2014117690A RU 2014117690 A RU2014117690 A RU 2014117690A
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Russia
Prior art keywords
lipid
particle
nucleic acid
interfering rna
composition
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RU2014117690/10A
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English (en)
Russian (ru)
Inventor
Иан МАКЛАХНЕН
Эми К.Х. ЛИ
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Протива Байотерапьютикс Инк.
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Application filed by Протива Байотерапьютикс Инк. filed Critical Протива Байотерапьютикс Инк.
Publication of RU2014117690A publication Critical patent/RU2014117690A/ru

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/30Drugs for disorders of the nervous system for treating abuse or dependence
    • A61P25/32Alcohol-abuse
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y102/00Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003Aldehyde 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)
  • 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)
RU2014117690/10A 2011-10-05 2012-10-04 Композиции и способы для сайленсинга альдегид-дегидрогеназы RU2014117690A (ru)

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
RU2014117690A true RU2014117690A (ru) 2015-11-10

Family

ID=48044157

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2014117690/10A RU2014117690A (ru) 2011-10-05 2012-10-04 Композиции и способы для сайленсинга альдегид-дегидрогеназы

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)

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RU2788030C2 (ru) * 2015-12-13 2023-01-16 Нитто Денко Корпорейшн Структуры мирнк с высокой активностью и сниженным воздействием вне мишени
US11926831B2 (en) 2015-12-13 2024-03-12 Nitto Denko Corporation SiRNA structures for high activity and reduced off target

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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
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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2788030C2 (ru) * 2015-12-13 2023-01-16 Нитто Денко Корпорейшн Структуры мирнк с высокой активностью и сниженным воздействием вне мишени
US11926831B2 (en) 2015-12-13 2024-03-12 Nitto Denko Corporation SiRNA structures for high activity and reduced off target

Also Published As

Publication number Publication date
AU2016201913A1 (en) 2016-04-21
AU2012318249A1 (en) 2013-05-02
CA2850792A1 (en) 2013-04-11
NZ623181A (en) 2015-11-27
AU2012318249B2 (en) 2016-04-21
HK1200837A1 (en) 2015-08-14
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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FA92 Acknowledgement of application withdrawn (lack of supplementary materials submitted)

Effective date: 20170111