JP7210287B2 - Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド - Google Patents

Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド Download PDF

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JP7210287B2
JP7210287B2 JP2018560776A JP2018560776A JP7210287B2 JP 7210287 B2 JP7210287 B2 JP 7210287B2 JP 2018560776 A JP2018560776 A JP 2018560776A JP 2018560776 A JP2018560776 A JP 2018560776A JP 7210287 B2 JP7210287 B2 JP 7210287B2
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alkyl
mrna
citrine
codon
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JP2019519511A5 (https=
JP2019519511A (ja
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パオロ・マルティーニ
スティーブン・ホージ
ケリー・ベネナート
ブラディミア・プレスニャク
イアン・マクファディエン
エララヘワゲ・サティヤジット・クマラシンゲ
ジンソン・ツァオ
リン・トゥン・グエイ
ステイシー・サブニス
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ModernaTx 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary 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
    • 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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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
JP2018560776A 2016-05-18 2017-05-18 Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド Active JP7210287B2 (ja)

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JP2023002608A JP2023055716A (ja) 2016-05-18 2023-01-11 Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド

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US201662338479P 2016-05-18 2016-05-18
US62/338,479 2016-05-18
PCT/US2017/033421 WO2017201349A1 (en) 2016-05-18 2017-05-18 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

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JP2019519511A5 JP2019519511A5 (https=) 2020-07-02
JP7210287B2 true JP7210287B2 (ja) 2023-01-23

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JP2023002608A Pending JP2023055716A (ja) 2016-05-18 2023-01-11 Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド

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US (2) US10993918B2 (https=)
EP (1) EP3458081B1 (https=)
JP (2) JP7210287B2 (https=)
AU (2) AU2017268396B2 (https=)
CA (1) CA3024625A1 (https=)
MA (1) MA45036A (https=)
MY (1) MY201498A (https=)
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Cited By (1)

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JP2022031359A (ja) * 2015-12-10 2022-02-18 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法

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