JP2016504050A - 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド - Google Patents

細胞表現型の改変のためのシグナルセンサーポリヌクレオチド Download PDF

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Publication number
JP2016504050A
JP2016504050A JP2015553717A JP2015553717A JP2016504050A JP 2016504050 A JP2016504050 A JP 2016504050A JP 2015553717 A JP2015553717 A JP 2015553717A JP 2015553717 A JP2015553717 A JP 2015553717A JP 2016504050 A JP2016504050 A JP 2016504050A
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Prior art keywords
cancer
signal sensor
mir
sensor polynucleotide
polynucleotide
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JP2015553717A
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Japanese (ja)
Inventor
ジー． ホーグ、スティーブン
ジー． ホーグ、スティーブン
チャクラボルティ、ティルタ
ピー． フレデリック、ジョシュア
ピー． フレデリック、ジョシュア
ジョン、マティアス
フジェローレ、アントニン ドゥ
フジェローレ、アントニン ドゥ
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Moderna Inc
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Moderna Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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]
    • C12N2310/141—MicroRNAs, miRNAs

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2015553717A 2013-01-17 2013-09-30 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド Pending JP2016504050A (ja)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201361753661P 2013-01-17 2013-01-17
US61/753,661 2013-01-17
US201361754159P 2013-01-18 2013-01-18
US61/754,159 2013-01-18
US201361781097P 2013-03-14 2013-03-14
US61/781,097 2013-03-14
US201361829334P 2013-05-31 2013-05-31
US61/829,334 2013-05-31
US201361839893P 2013-06-27 2013-06-27
US61/839,893 2013-06-27
US201361842733P 2013-07-03 2013-07-03
US61/842,733 2013-07-03
US201361857304P 2013-07-23 2013-07-23
US61/857,304 2013-07-23
PCT/US2013/062531 WO2014113089A2 (en) 2013-01-17 2013-09-30 Signal-sensor polynucleotides for the alteration of cellular phenotypes

Related Child Applications (1)

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JP2017016882A Division JP2017113010A (ja) 2013-01-17 2017-02-01 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド

Publications (1)

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JP2016504050A true JP2016504050A (ja) 2016-02-12

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JP2015553717A Pending JP2016504050A (ja) 2013-01-17 2013-09-30 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド
JP2017016882A Pending JP2017113010A (ja) 2013-01-17 2017-02-01 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド
JP2018146351A Active JP6726711B2 (ja) 2013-01-17 2018-08-03 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド

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JP2018146351A Active JP6726711B2 (ja) 2013-01-17 2018-08-03 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド

Country Status (7)

Country Link
US (4) US20140200261A1 (enExample)
EP (2) EP2946014A2 (enExample)
JP (3) JP2016504050A (enExample)
AU (2) AU2013374345A1 (enExample)
CA (1) CA2897941A1 (enExample)
HK (1) HK1217215A1 (enExample)
WO (1) WO2014113089A2 (enExample)

Cited By (10)

* Cited by examiner, † Cited by third party
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JP2017113010A (ja) * 2013-01-17 2017-06-29 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド
KR20180131577A (ko) * 2016-03-31 2018-12-10 에트리스 게엠베하 신규의 최소 utr 서열
JP2019516710A (ja) * 2016-05-18 2019-06-20 モデルナティーエックス， インコーポレイテッド インターロイキン−１２（ｉｌ１２）をコードするポリヌクレオチドおよびその使用
JP2019519516A (ja) * 2016-05-18 2019-07-11 モデルナティーエックス， インコーポレイテッド がんの治療のためのｍＲＮＡ併用療法
JP2019519517A (ja) * 2016-05-18 2019-07-11 モデルナティーエックス， インコーポレイテッド 免疫調節ポリペプチドをコードするｍＲＮＡの組み合わせ及びその使用
JPWO2019013294A1 (ja) * 2017-07-12 2020-10-22 国立大学法人京都大学 高発現性ｍＲＮＡスイッチ
US10925935B2 (en) 2012-11-26 2021-02-23 Modernatx, Inc. Terminally Modified RNA
JP2021534778A (ja) * 2018-08-30 2021-12-16 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル フィードバック使用可能合成遺伝子、標的シードマッチカセット、およびその使用
JP2022136230A (ja) * 2016-05-18 2022-09-15 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
KR102913044B1 (ko) 2018-08-30 2026-01-16 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 피드백이 가능한 합성 유전자, 표적 시드 매치 카세트, 및 그 용도

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