JP2015518705A - ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド - Google Patents

ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド Download PDF

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Publication number
JP2015518705A
JP2015518705A JP2015504565A JP2015504565A JP2015518705A JP 2015518705 A JP2015518705 A JP 2015518705A JP 2015504565 A JP2015504565 A JP 2015504565A JP 2015504565 A JP2015504565 A JP 2015504565A JP 2015518705 A JP2015518705 A JP 2015518705A
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JP
Japan
Prior art keywords
optionally substituted
independently
polynucleotide
alkyl
polypeptide
Prior art date
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Pending
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JP2015504565A
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English (en)
Japanese (ja)
Inventor
バンセル、ステファヌ
チャクラボルティ、ティルタ
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
エム． エルバシール、セイダ
エム． エルバシール、セイダ
ジョン、マティアス
ロイ、アタヌ
ホリスキー、スーザン
エム． ウッド、クリスティ
エム． ウッド、クリスティ
ハタラ、ポール
ピー． シュラム、ジェイソン
ピー． シュラム、ジェイソン
エジェベ、ケネチ
リン エルスワース、ジェフ
リン エルスワース、ジェフ
ギルド、ジャスティン
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Moderna Inc
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Moderna Therapeutics Inc
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Application filed by Moderna Therapeutics Inc filed Critical Moderna Therapeutics Inc
Publication of JP2015518705A publication Critical patent/JP2015518705A/ja
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • 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
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Reproductive Health (AREA)
JP2015504565A 2012-04-02 2013-03-09 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド Pending JP2015518705A (ja)

Applications Claiming Priority (101)

Application Number Priority Date Filing Date Title
US201261618957P 2012-04-02 2012-04-02
US201261618896P 2012-04-02 2012-04-02
US201261618953P 2012-04-02 2012-04-02
US201261618866P 2012-04-02 2012-04-02
US201261618961P 2012-04-02 2012-04-02
US201261618878P 2012-04-02 2012-04-02
US201261618868P 2012-04-02 2012-04-02
US201261618945P 2012-04-02 2012-04-02
US201261618870P 2012-04-02 2012-04-02
US201261618911P 2012-04-02 2012-04-02
US201261618862P 2012-04-02 2012-04-02
US201261618885P 2012-04-02 2012-04-02
US201261618873P 2012-04-02 2012-04-02
US201261618922P 2012-04-02 2012-04-02
US61/618,945 2012-04-02
US61/618,961 2012-04-02
US61/618,862 2012-04-02
US61/618,911 2012-04-02
US61/618,866 2012-04-02
US61/618,896 2012-04-02
US61/618,953 2012-04-02
US61/618,922 2012-04-02
US61/618,878 2012-04-02
US61/618,957 2012-04-02
US61/618,868 2012-04-02
US61/618,885 2012-04-02
US61/618,870 2012-04-02
US61/618,873 2012-04-02
US201261648286P 2012-05-17 2012-05-17
US201261648244P 2012-05-17 2012-05-17
US61/648,286 2012-05-17
US61/648,244 2012-05-17
US201261668157P 2012-07-05 2012-07-05
US61/668,157 2012-07-05
US201261681720P 2012-08-10 2012-08-10
US201261681667P 2012-08-10 2012-08-10
US201261681687P 2012-08-10 2012-08-10
US201261681742P 2012-08-10 2012-08-10
US201261681645P 2012-08-10 2012-08-10
US201261681647P 2012-08-10 2012-08-10
US201261681712P 2012-08-10 2012-08-10
US201261681696P 2012-08-10 2012-08-10
US201261681654P 2012-08-10 2012-08-10
US201261681649P 2012-08-10 2012-08-10
US201261681648P 2012-08-10 2012-08-10
US201261681661P 2012-08-10 2012-08-10
US201261681704P 2012-08-10 2012-08-10
US201261681675P 2012-08-10 2012-08-10
US201261681658P 2012-08-10 2012-08-10
US201261681650P 2012-08-10 2012-08-10
US61/681,654 2012-08-10
US61/681,648 2012-08-10
US61/681,650 2012-08-10
US61/681,687 2012-08-10
US61/681,675 2012-08-10
US61/681,696 2012-08-10
US61/681,658 2012-08-10
US61/681,742 2012-08-10
US61/681,647 2012-08-10
US61/681,704 2012-08-10
US61/681,712 2012-08-10
US61/681,645 2012-08-10
US61/681,667 2012-08-10
US61/681,661 2012-08-10
US61/681,649 2012-08-10
US61/681,720 2012-08-10
US201261696381P 2012-09-04 2012-09-04
US61/696,381 2012-09-04
US201261709303P 2012-10-03 2012-10-03
US61/709,303 2012-10-03
US201261712490P 2012-10-11 2012-10-11
US61/712,490 2012-10-11
US201261737213P 2012-12-14 2012-12-14
US201261737174P 2012-12-14 2012-12-14
US201261737155P 2012-12-14 2012-12-14
US201261737203P 2012-12-14 2012-12-14
US201261737130P 2012-12-14 2012-12-14
US201261737168P 2012-12-14 2012-12-14
US201261737160P 2012-12-14 2012-12-14
US201261737152P 2012-12-14 2012-12-14
US201261737135P 2012-12-14 2012-12-14
US201261737139P 2012-12-14 2012-12-14
US201261737147P 2012-12-14 2012-12-14
US201261737184P 2012-12-14 2012-12-14
US201261737191P 2012-12-14 2012-12-14
US201261737134P 2012-12-14 2012-12-14
US61/737,139 2012-12-14
US61/737,152 2012-12-14
US61/737,203 2012-12-14
US61/737,174 2012-12-14
US61/737,184 2012-12-14
US61/737,160 2012-12-14
US61/737,155 2012-12-14
US61/737,134 2012-12-14
US61/737,191 2012-12-14
US61/737,130 2012-12-14
US61/737,135 2012-12-14
US61/737,147 2012-12-14
US61/737,213 2012-12-14
US61/737,168 2012-12-14
PCT/US2013/030062 WO2013151666A2 (en) 2012-04-02 2013-03-09 Modified polynucleotides for the production of biologics and proteins associated with human disease

Related Child Applications (1)

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JP2017018370A Division JP6430552B2 (ja) 2012-04-02 2017-02-03 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド

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JP2015518705A true JP2015518705A (ja) 2015-07-06

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Family Applications (5)

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JP2015504565A Pending JP2015518705A (ja) 2012-04-02 2013-03-09 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2017018370A Active JP6430552B2 (ja) 2012-04-02 2017-02-03 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2018204593A Active JP6921797B2 (ja) 2012-04-02 2018-10-31 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2021122947A Pending JP2021192606A (ja) 2012-04-02 2021-07-28 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2023114173A Pending JP2023130471A (ja) 2012-04-02 2023-07-12 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド

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JP2017018370A Active JP6430552B2 (ja) 2012-04-02 2017-02-03 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2018204593A Active JP6921797B2 (ja) 2012-04-02 2018-10-31 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2021122947A Pending JP2021192606A (ja) 2012-04-02 2021-07-28 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
JP2023114173A Pending JP2023130471A (ja) 2012-04-02 2023-07-12 ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド

Country Status (6)

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JP (5) JP2015518705A (xx)
CN (2) CN108949772A (xx)
AU (4) AU2013243949A1 (xx)
CA (1) CA2868391A1 (xx)
HK (1) HK1206612A1 (xx)
WO (1) WO2013151666A2 (xx)

Cited By (9)

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JP6231252B1 (ja) * 2016-07-08 2017-11-15 花王株式会社 核酸試料の調製方法
WO2018008319A1 (ja) * 2016-07-08 2018-01-11 花王株式会社 核酸試料の調製方法
JP2019500430A (ja) * 2015-12-17 2019-01-10 モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． メチルマロニルＣｏＡムターゼをコードするポリヌクレオチド
JP2019533708A (ja) * 2016-11-10 2019-11-21 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の皮下送達
JP2020061955A (ja) * 2018-10-16 2020-04-23 公立大学法人横浜市立大学 新生児期〜小児期発症の脳小血管病又はその保因者の検出方法
JP2021504343A (ja) * 2017-11-22 2021-02-15 モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
JP2021052808A (ja) * 2013-03-14 2021-04-08 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ｍＲＮＡによってコードされる抗体を送達するための方法及び組成物
JP2021523916A (ja) * 2018-05-15 2021-09-09 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の皮下送達
US11739345B2 (en) 2018-05-09 2023-08-29 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria

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