JP2019519601A - 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド - Google Patents

急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド Download PDF

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JP2019519601A
JP2019519601A JP2019512957A JP2019512957A JP2019519601A JP 2019519601 A JP2019519601 A JP 2019519601A JP 2019512957 A JP2019512957 A JP 2019512957A JP 2019512957 A JP2019512957 A JP 2019512957A JP 2019519601 A JP2019519601 A JP 2019519601A
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pharmaceutical composition
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polynucleotide
pbgd
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JP2019519601A5 (enExample
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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
レイ・ジャン
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
アントニオ・フォンタネラス・ロマ
ペドロ・ベラオンド・ロペス
マティアス・アントニオ・アビラ・サラゴサ
リン・トゥン・グエイ
ステイシー・サブニス
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Fundacion para la Investigacion Medica Aplicada
ModernaTx Inc
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Fundacion para la Investigacion Medica Aplicada
ModernaTx Inc
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Priority claimed from PCT/US2017/033418 external-priority patent/WO2017201346A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/08—Plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for electrolytic or acid-base disorders, e.g. hypovolemic shock
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/01061—Hydroxymethylbilane synthase (2.5.1.61)

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  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)
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JP2019512957A 2016-05-18 2017-05-18 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド Pending JP2019519601A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023023253A JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338161P 2016-05-18 2016-05-18
US62/338,161 2016-05-18
EP17382259 2017-05-09
EP17382259.4 2017-05-09
PCT/US2017/033418 WO2017201346A1 (en) 2016-05-18 2017-05-18 Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria

Related Child Applications (1)

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JP2023023253A Division JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

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JP2019519601A true JP2019519601A (ja) 2019-07-11
JP2019519601A5 JP2019519601A5 (enExample) 2020-06-25

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JP2023023253A Pending JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

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JP (2) JP2019519601A (enExample)
MA (1) MA45032A (enExample)

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JP2023520047A (ja) * 2020-04-01 2023-05-15 トランスレイト バイオ， インコーポレイテッド フェノール酸脂質系カチオン性脂質
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

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Cited By (6)

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Publication number Priority date Publication date Assignee Title
JP2023021468A (ja) * 2017-03-15 2023-02-13 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物及び組成物
JP7596343B2 (ja) 2017-03-15 2024-12-09 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物及び組成物
JP2023520047A (ja) * 2020-04-01 2023-05-15 トランスレイト バイオ， インコーポレイテッド フェノール酸脂質系カチオン性脂質
JP7791103B2 (ja) 2020-04-01 2025-12-23 トランスレイト バイオ， インコーポレイテッド フェノール酸脂質系カチオン性脂質
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom
US12576040B2 (en) 2020-10-14 2026-03-17 The Trustees Of The University Of Pennsylvania Ionizable lipids and methods of manufacture and use thereof

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