JP7832114B2 - オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法 - Google Patents

オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法

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Publication number
JP7832114B2
JP7832114B2 JP2022552798A JP2022552798A JP7832114B2 JP 7832114 B2 JP7832114 B2 JP 7832114B2 JP 2022552798 A JP2022552798 A JP 2022552798A JP 2022552798 A JP2022552798 A JP 2022552798A JP 7832114 B2 JP7832114 B2 JP 7832114B2
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mrna
lipid
sequence
mol
otc
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Japanese (ja)
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JPWO2021178510A5 (https=
JP2023516676A5 (https=
JP2023516676A (ja
Inventor
チブクラ，パドマナブ
プラカシュ カルマリ，プリヤ
清 立川
イー パーカー，スザンヌ
ロドリゲス サブラード，マルチャーノ
リンフォン，パトララニー
バオ，ヤンジエ
ボイド リー ベガ，ジェレル
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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    • 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
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • A61K31/22—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin
    • A61K31/221—Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids of acyclic acids, e.g. pravastatin with compounds having an amino group, e.g. acetylcholine, acetylcarnitine
    • 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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Emergency Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
JP2022552798A 2020-03-03 2021-03-03 オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法 Active JP7832114B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062984764P 2020-03-03 2020-03-03
US62/984,764 2020-03-03
PCT/US2021/020634 WO2021178510A1 (en) 2020-03-03 2021-03-03 Compositions and methods for the treatment of ornithine transcarbamylase deficiency

Publications (4)

Publication Number Publication Date
JP2023516676A JP2023516676A (ja) 2023-04-20
JPWO2021178510A5 JPWO2021178510A5 (https=) 2024-03-12
JP2023516676A5 JP2023516676A5 (https=) 2024-03-12
JP7832114B2 true JP7832114B2 (ja) 2026-03-17

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Country Status (6)

Country Link
US (2) US12351834B2 (https=)
EP (1) EP4114470A4 (https=)
JP (1) JP7832114B2 (https=)
AU (1) AU2021231795A1 (https=)
CA (1) CA3169889A1 (https=)
WO (1) WO2021178510A1 (https=)

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JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
WO2021183563A1 (en) 2020-03-09 2021-09-16 Arcturus Therapeutics, Inc. Coronavirus vaccine compositions and methods
US12178921B2 (en) 2020-08-14 2024-12-31 Arcturus Therapeutics, Inc. Method of lyophilizing lipid nanoparticles
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
EP4328313A4 (en) * 2021-04-21 2025-06-25 Jichi Medical University Adeno-associated virus virion for treating ornithine transcarbamylase deficiency
US20250041238A1 (en) * 2021-12-23 2025-02-06 Etherna Immunotherapies Nv METHOD FOR LAGRE-SCALE PRODUCTION OF LARGE-SIZE LNPs
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
CN116087165B (zh) * 2022-12-22 2023-07-28 南京林业大学 一种简单快速检测mtc和/或otc和/或tc的方法
WO2025053636A1 (ko) * 2023-09-05 2025-03-13 한국생명공학연구원 지질 나노 입자를 이용한 핵산 전달 시스템

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Also Published As

Publication number Publication date
US20210284974A1 (en) 2021-09-16
US20250136953A1 (en) 2025-05-01
AU2021231795A1 (en) 2022-10-06
CA3169889A1 (en) 2021-09-10
WO2021178510A1 (en) 2021-09-10
EP4114470A1 (en) 2023-01-11
US12351834B2 (en) 2025-07-08
JP2023516676A (ja) 2023-04-20
EP4114470A4 (en) 2024-04-17

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