JP2019528284A - 核酸産物およびその投与方法 - Google Patents

核酸産物およびその投与方法 Download PDF

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JP2019528284A
JP2019528284A JP2019509510A JP2019509510A JP2019528284A JP 2019528284 A JP2019528284 A JP 2019528284A JP 2019509510 A JP2019509510 A JP 2019509510A JP 2019509510 A JP2019509510 A JP 2019509510A JP 2019528284 A JP2019528284 A JP 2019528284A
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disease
defective gene
appropriate
gene
synthetic rna
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JP2019528284A5 (OSRAM
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エンジェル，マシュー
ローデ，クリストファー
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ファクター バイオサイエンス インコーポレイテッド
ファクター バイオサイエンス インコーポレイテッド
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Publication of JP2019528284A publication Critical patent/JP2019528284A/ja
Publication of JP2019528284A5 publication Critical patent/JP2019528284A5/ja
Priority to JP2022168829A priority Critical patent/JP2023011696A/ja
Priority to JP2022168830A priority patent/JP2023011697A/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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P3/06—Antihyperlipidemics
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07—ORGANIC CHEMISTRY
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    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)

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JP2019509510A 2016-08-17 2017-08-17 核酸産物およびその投与方法 Pending JP2019528284A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022168829A JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168830A JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662376209P 2016-08-17 2016-08-17
US62/376,209 2016-08-17
US201762509350P 2017-05-22 2017-05-22
US62/509,350 2017-05-22
PCT/US2017/047440 WO2018035377A1 (en) 2016-08-17 2017-08-17 Nucleic acid products and methods of administration thereof

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JP2022168830A Division JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168829A Division JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

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JP2019528284A5 JP2019528284A5 (OSRAM) 2020-09-24

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JP2022168830A Pending JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
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US (12) US10576167B2 (OSRAM)
EP (1) EP3500585A4 (OSRAM)
JP (3) JP2019528284A (OSRAM)
CN (2) CN116115629A (OSRAM)
AU (2) AU2017312113B2 (OSRAM)
CA (1) CA3033788A1 (OSRAM)
IL (2) IL308824A (OSRAM)
WO (1) WO2018035377A1 (OSRAM)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023011696A (ja) * 2016-08-17 2023-01-24 ファクター バイオサイエンス インコーポレイテッド 核酸産物およびその投与方法
JP2023503636A (ja) * 2019-11-26 2023-01-31 コリア リサーチ インスティテュート オブ バイオサイエンス アンド バイオテクノロジー タンパク質発現用ｍＲＮＡ構造体及びその用途

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