JP2014530602A - アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法 - Google Patents

アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法 Download PDF

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JP2014530602A
JP2014530602A JP2014534724A JP2014534724A JP2014530602A JP 2014530602 A JP2014530602 A JP 2014530602A JP 2014534724 A JP2014534724 A JP 2014534724A JP 2014534724 A JP2014534724 A JP 2014534724A JP 2014530602 A JP2014530602 A JP 2014530602A
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lipid
mol
nucleic acid
nucleotides
particle
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JP2014530602A5 (cg-RX-API-DMAC7.html
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イアン マクラックラン，
イアン マクラックラン，
アミー シー．エイチ． リー，
アミー シー．エイチ． リー，
Original Assignee
プロティバ バイオセラピューティクス インコーポレイテッド
プロティバ バイオセラピューティクス インコーポレイテッド
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Publication of JP2014530602A publication Critical patent/JP2014530602A/ja
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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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Addiction (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)
JP2014534724A 2011-10-05 2012-10-04 アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法 Pending JP2014530602A (ja)

Applications Claiming Priority (4)

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

Publications (2)

Publication Number Publication Date
JP2014530602A true JP2014530602A (ja) 2014-11-20
JP2014530602A5 JP2014530602A5 (cg-RX-API-DMAC7.html) 2015-09-10

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JP2014534724A Pending JP2014530602A (ja) 2011-10-05 2012-10-04 アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法

Country Status (10)

Country Link
US (1) US8999950B2 (cg-RX-API-DMAC7.html)
EP (1) EP2764007A4 (cg-RX-API-DMAC7.html)
JP (1) JP2014530602A (cg-RX-API-DMAC7.html)
KR (1) KR20140097143A (cg-RX-API-DMAC7.html)
CN (1) CN104114571A (cg-RX-API-DMAC7.html)
AU (2) AU2012318249B2 (cg-RX-API-DMAC7.html)
CA (1) CA2850792A1 (cg-RX-API-DMAC7.html)
HK (1) HK1200837A1 (cg-RX-API-DMAC7.html)
RU (1) RU2014117690A (cg-RX-API-DMAC7.html)
WO (1) WO2013052677A1 (cg-RX-API-DMAC7.html)

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JP2021511042A (ja) * 2018-01-16 2021-05-06 ディセルナ ファーマシューティカルズ インコーポレイテッド Ａｌｄｈ２発現を阻害するための組成物及び方法
JP2021517826A (ja) * 2018-03-02 2021-07-29 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｇｙｓ２発現を阻害するための組成物及び方法

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JP5475753B2 (ja) 2008-04-15 2014-04-16 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 核酸送達用の脂質製剤
CA2767127A1 (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
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
CA3198966A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Cleavable lipids
EP2717893B1 (en) 2011-06-08 2019-05-08 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
CA2868034C (en) 2012-03-29 2021-07-27 Shire Human Genetic Therapies, Inc. Ionizable cationic lipids
HK1206644A1 (en) 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2013185069A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Pulmonary delivery of mrna to non-lung target cells
TR201901310T4 (tr) 2013-03-14 2019-02-21 Translate Bio Inc Mesajcı RNA'nın saflaştırılması yöntemleri.
LT2968586T (lt) 2013-03-14 2018-11-26 Translate Bio, Inc. Cft mrnr kompozicijos bei su jomis susiję būdai ir panaudojimai
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
CN112618732A (zh) 2013-10-22 2021-04-09 夏尔人类遗传性治疗公司 用于递送信使rna的脂质制剂
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
CN105658800A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 Mrna的cns递送及其用途
KR102739894B1 (ko) 2014-04-25 2024-12-05 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
US10022455B2 (en) 2014-05-30 2018-07-17 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
CN111588695A (zh) 2014-06-24 2020-08-28 川斯勒佰尔公司 用于递送核酸的立体化学富集组合物
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
AU2016233135B2 (en) 2015-03-19 2021-07-08 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
CA3001852A1 (en) 2015-10-14 2017-04-20 Translate Bio, Inc. Modification of rna-related enzymes for enhanced production
US10358647B2 (en) 2015-12-13 2019-07-23 Nitto Denko Corporation siRNA structures for high activity and reduced off target
EP3585417B1 (en) 2017-02-27 2023-02-22 Translate Bio, Inc. Method of making a codon-optimized cftr mrna
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
EP3735461A1 (en) * 2017-11-13 2020-11-11 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of aldh2 in a cell
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
EP4379053A3 (en) 2018-10-09 2024-09-18 The University of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
CA3120647A1 (en) 2018-11-21 2020-05-28 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

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JP2010507680A (ja) * 2006-10-24 2010-03-11 サーナ セラピューティクス インコーポレーテッド 生物学的に活性な分子を送達するための脂質ナノ粒子系組成物及び方法
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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021511042A (ja) * 2018-01-16 2021-05-06 ディセルナ ファーマシューティカルズ インコーポレイテッド Ａｌｄｈ２発現を阻害するための組成物及び方法
JP2021517826A (ja) * 2018-03-02 2021-07-29 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｇｙｓ２発現を阻害するための組成物及び方法
JP7453921B2 (ja) 2018-03-02 2024-03-21 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｇｙｓ２発現を阻害するための組成物及び方法

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Publication number Publication date
NZ623181A (en) 2015-11-27
CN104114571A (zh) 2014-10-22
EP2764007A4 (en) 2015-05-06
KR20140097143A (ko) 2014-08-06
AU2012318249A1 (en) 2013-05-02
CA2850792A1 (en) 2013-04-11
RU2014117690A (ru) 2015-11-10
AU2012318249B2 (en) 2016-04-21
US8999950B2 (en) 2015-04-07
WO2013052677A1 (en) 2013-04-11
HK1200837A1 (en) 2015-08-14
AU2016201913A1 (en) 2016-04-21
US20140248338A1 (en) 2014-09-04
EP2764007A1 (en) 2014-08-13

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