CN116096428A - 鸟氨酸氨甲酰基转移酶(otc)构建体及其使用方法 - Google Patents

鸟氨酸氨甲酰基转移酶(otc)构建体及其使用方法 Download PDF

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Publication number
CN116096428A
CN116096428A CN202080089266.1A CN202080089266A CN116096428A CN 116096428 A CN116096428 A CN 116096428A CN 202080089266 A CN202080089266 A CN 202080089266A CN 116096428 A CN116096428 A CN 116096428A
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Prior art keywords
otc
polynucleotide construct
mrna
lnp
sequence
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Chinese (zh)
Inventor
M·普雷威
O·戴利
K·拉姆
J·海斯
R·霍尔兰德
C·伊扫
E·亚沃尔斯基
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Gainawan Science Co ltd
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Gainawan Science Co ltd
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Publication of CN116096428A publication Critical patent/CN116096428A/zh
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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
    • 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
    • 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
    • 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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    • 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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    • C12N15/00—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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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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    • 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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  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
CN202080089266.1A 2019-10-22 2020-10-22 鸟氨酸氨甲酰基转移酶(otc)构建体及其使用方法 Pending CN116096428A (zh)

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

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CN116096428A true CN116096428A (zh) 2023-05-09

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CN202080089266.1A Pending CN116096428A (zh) 2019-10-22 2020-10-22 鸟氨酸氨甲酰基转移酶(otc)构建体及其使用方法

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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
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
WO2022229903A1 (en) * 2021-04-28 2022-11-03 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
KR20250169641A (ko) * 2023-03-06 2025-12-03 제네럴 메디신스 엘엘씨 코돈 최적화 및 이의 사용 방법

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160206750A1 (en) * 2013-07-30 2016-07-21 Phaserx, Inic. Block copolymers
US20180221402A1 (en) * 2015-01-21 2018-08-09 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
WO2019104152A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
WO2015011633A1 (en) * 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
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
CA3027312A1 (en) * 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
FI3568483T3 (fi) * 2017-01-10 2025-11-21 Childrens Medical Res Institute Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160206750A1 (en) * 2013-07-30 2016-07-21 Phaserx, Inic. Block copolymers
US20180221402A1 (en) * 2015-01-21 2018-08-09 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
WO2019104152A1 (en) * 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

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Publication number Publication date
WO2021081225A1 (en) 2021-04-29
US20230001021A1 (en) 2023-01-05
KR20240130161A (ko) 2024-08-29
JP2022553375A (ja) 2022-12-22
CA3158626A1 (en) 2021-04-29
EP4048317A4 (en) 2023-11-22
IL292420A (en) 2022-06-01
AU2020371697A1 (en) 2022-05-26
EP4048317A1 (en) 2022-08-31
JP7826197B2 (ja) 2026-03-09

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