KR20240130161A - 오르니틴 트랜스카르바밀라제 (otc) 구축물 및 그의 사용 방법 - Google Patents

오르니틴 트랜스카르바밀라제 (otc) 구축물 및 그의 사용 방법 Download PDF

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KR20240130161A
KR20240130161A KR1020227017024A KR20227017024A KR20240130161A KR 20240130161 A KR20240130161 A KR 20240130161A KR 1020227017024 A KR1020227017024 A KR 1020227017024A KR 20227017024 A KR20227017024 A KR 20227017024A KR 20240130161 A KR20240130161 A KR 20240130161A
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South Korea
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otc
polynucleotide construct
sequence
mrna
composition
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Korean (ko)
Inventor
메리 프리브
오웬 달리
키유 램
제임스 헤이어스
리차드 홀랜드
크리스틴 에사우
에드 야워스키
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제네반트 사이언시즈 게엠베하
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Publication of KR20240130161A publication Critical patent/KR20240130161A/ko
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    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • 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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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
KR1020227017024A 2019-10-22 2020-10-22 오르니틴 트랜스카르바밀라제 (otc) 구축물 및 그의 사용 방법 Pending KR20240130161A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924567P 2019-10-22 2019-10-22
US62/924,567 2019-10-22
PCT/US2020/056890 WO2021081225A1 (en) 2019-10-22 2020-10-22 Ornithine transcarbamylase (otc) constructs and methods of using the same

Publications (1)

Publication Number Publication Date
KR20240130161A true KR20240130161A (ko) 2024-08-29

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KR1020227017024A Pending KR20240130161A (ko) 2019-10-22 2020-10-22 오르니틴 트랜스카르바밀라제 (otc) 구축물 및 그의 사용 방법

Country Status (9)

Country Link
US (1) US20230001021A1 (https=)
EP (1) EP4048317A4 (https=)
JP (1) JP7826197B2 (https=)
KR (1) KR20240130161A (https=)
CN (1) CN116096428A (https=)
AU (1) AU2020371697A1 (https=)
CA (1) CA3158626A1 (https=)
IL (1) IL292420A (https=)
WO (1) WO2021081225A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
US20240216545A1 (en) * 2021-04-28 2024-07-04 Genevant Sciences Gmbh Mrna delivery constructs and methods of using the same
KR20230083893A (ko) * 2021-12-03 2023-06-12 고려대학교 산학협력단 번역 효율이 향상된 5'-utr, 이를 포함하는 합성 핵산 분자 및 이를 포함하는 백신 또는 치료제 조성물
CN121335707A (zh) * 2023-03-06 2026-01-13 通用药品有限责任公司 密码子优化及其使用方法

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2817287B1 (en) 2012-02-24 2018-10-03 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
JP6620093B2 (ja) * 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
AU2014296278C1 (en) * 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
US9765477B2 (en) * 2013-08-23 2017-09-19 Dow Global Technologies Llc Low density attached polyurethane foams made using a combination of frothing and blowing methods
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
EP3469074B1 (en) * 2016-06-13 2020-12-09 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
PT3568483T (pt) * 2017-01-10 2025-11-18 Childrens Medical Res Institute Polinucleótidos e vetores para a expressão de transgenes
CA3079428A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

Also Published As

Publication number Publication date
WO2021081225A1 (en) 2021-04-29
JP2022553375A (ja) 2022-12-22
EP4048317A1 (en) 2022-08-31
AU2020371697A1 (en) 2022-05-26
US20230001021A1 (en) 2023-01-05
CA3158626A1 (en) 2021-04-29
IL292420A (en) 2022-06-01
EP4048317A4 (en) 2023-11-22
CN116096428A (zh) 2023-05-09
JP7826197B2 (ja) 2026-03-09

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