JP2019516679A - 生物活性剤に関する方法および組成物 - Google Patents

生物活性剤に関する方法および組成物 Download PDF

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JP2019516679A
JP2019516679A JP2018557083A JP2018557083A JP2019516679A JP 2019516679 A JP2019516679 A JP 2019516679A JP 2018557083 A JP2018557083 A JP 2018557083A JP 2018557083 A JP2018557083 A JP 2018557083A JP 2019516679 A JP2019516679 A JP 2019516679A
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hydrochloride
muscle
composition
oligonucleotide
lipid
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Japanese (ja)
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チャンドラ ヴァージェシー
チャンドラ ヴァージェシー
ジェイソン ジンシン チャン
ジェイソン ジンシン チャン
セスマダバン ディバカラメノン
セスマダバン ディバカラメノン
デビット チャールズ ドネル バトラー
デビット チャールズ ドネル バトラー
ゲンリャン ル
ゲンリャン ル
ナオキ イワモト
ナオキ イワモト
ハイリン ヤン
ハイリン ヤン
マリア デイビッド フランク−カメネスキー
マリア デイビッド フランク−カメネスキー
スブラマニアン マラッパン
スブラマニアン マラッパン
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication of JP2019516679A publication Critical patent/JP2019516679A/ja
Priority to JP2023041621A priority Critical patent/JP2023078293A/ja
Priority to JP2025064806A priority patent/JP2025108550A/ja
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • 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]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/16—Aptamers
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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JP2018557083A 2016-05-04 2017-05-03 生物活性剤に関する方法および組成物 Pending JP2019516679A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023041621A JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物
JP2025064806A JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662331961P 2016-05-04 2016-05-04
US62/331,961 2016-05-04
US201662405810P 2016-10-07 2016-10-07
US62/405,810 2016-10-07
PCT/US2017/030777 WO2017192679A1 (en) 2016-05-04 2017-05-03 Methods and compositions of biologically active agents

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JP2023041621A Pending JP2023078293A (ja) 2016-05-04 2023-03-16 生物活性剤に関する方法および組成物
JP2025064806A Pending JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

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JP2025064806A Pending JP2025108550A (ja) 2016-05-04 2025-04-10 生物活性剤に関する方法および組成物

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US (2) US20190249173A1 (enExample)
EP (1) EP3452597A4 (enExample)
JP (3) JP2019516679A (enExample)
CN (1) CN109462995A (enExample)
MA (1) MA45290A (enExample)
WO (1) WO2017192679A1 (enExample)

Cited By (2)

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JP2023503644A (ja) * 2019-11-27 2023-01-31 ディー・ティー・エックス・ファーマ・インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための化合物および方法
JP2023545041A (ja) * 2020-10-05 2023-10-26 エスキュイグレック・テラピュティク Ｄｍｄ患者におけるエクソン５１スキッピングによるジストロフィンレスキューのためのオリゴマー化合物

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