JP2023532663A - 環状rna翻訳を駆動する遺伝エレメント及び使用方法 - Google Patents

環状rna翻訳を駆動する遺伝エレメント及び使用方法 Download PDF

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JP2023532663A
JP2023532663A JP2022580043A JP2022580043A JP2023532663A JP 2023532663 A JP2023532663 A JP 2023532663A JP 2022580043 A JP2022580043 A JP 2022580043A JP 2022580043 A JP2022580043 A JP 2022580043A JP 2023532663 A JP2023532663 A JP 2023532663A
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ires
sequence
protein
nucleic acid
circular rna
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JP2023532663A5 (https=
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チャン,ハワード・ワイ
チェン,チュン-カン
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Leland Stanford Junior University
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2022580043A 2020-06-25 2021-06-25 環状rna翻訳を駆動する遺伝エレメント及び使用方法 Pending JP2023532663A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063043964P 2020-06-25 2020-06-25
US63/043,964 2020-06-25
US202163186507P 2021-05-10 2021-05-10
US63/186,507 2021-05-10
PCT/US2021/039127 WO2021263124A2 (en) 2020-06-25 2021-06-25 Genetic elements driving circular rna translation and methods of use

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JP2023532663A true JP2023532663A (ja) 2023-07-31
JP2023532663A5 JP2023532663A5 (https=) 2024-07-02

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US (3) US11560567B2 (https=)
EP (1) EP4171631A4 (https=)
JP (1) JP2023532663A (https=)
KR (1) KR20230028375A (https=)
CN (1) CN116323945A (https=)
AU (1) AU2021297340A1 (https=)
CA (1) CA3180224A1 (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025520771A (ja) * 2022-06-29 2025-07-03 ソウル大学校産学協力団 RNAの安定性又はmRNA翻訳増強用の新規調節エレメントおよびこれと相互作用するZCCHC2、並びにその用途

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240299520A1 (en) * 2021-02-09 2024-09-12 Virginia Commonwealth University Mini circular rna therapeutics and vaccines and methods of use thereof
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
EP4436984A1 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Coronavirus immunogen compositions and their uses
CN118401544A (zh) 2021-11-24 2024-07-26 旗舰创业创新六公司 水痘-带状疱疹病毒免疫原组合物及其用途
KR20240118174A (ko) 2021-12-17 2024-08-02 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변성 조건 하에서의 원형 rna의 풍부화를 위한 방법
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
KR20240118881A (ko) 2021-12-23 2024-08-05 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 항푸소제닉 폴리펩티드를 인코딩하는 원형 폴리리보뉴클레오티드
CN119095967A (zh) * 2022-02-24 2024-12-06 普罗拜奥根股份公司 病毒样颗粒介导的环状rna递送
EP4493197A4 (en) * 2022-03-17 2026-03-18 Univ Leland Stanford Junior COMPOSITIONS AND METHODS FOR ENHANCED PROTEIN TRANSLATION FROM RECOMBED CIRCULAR RNA
US20250320519A1 (en) 2022-05-30 2025-10-16 Shanghai Circode Biomed Co., Ltd. Synthetic circular rna compositions and methods of use thereof
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状rna分子を調製するための組成物及び方法
US20260002223A1 (en) * 2022-06-27 2026-01-01 Wisconsin Alumni Research Foundation Universal method for parasite and eukaryotic endosymbiont identification
WO2024010028A1 (ja) * 2022-07-06 2024-01-11 国立大学法人京都大学 環状rna分子及びこれを用いた翻訳制御方法、翻訳活性化システム、並びに医薬組成物
CN115394376B (zh) * 2022-09-28 2023-04-18 奥明(杭州)生物医药有限公司 一种预测环状rna ires的方法
TW202434728A (zh) 2022-11-08 2024-09-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸的組成物和方法
KR20250122519A (ko) 2022-12-22 2025-08-13 젠스크립트 유에스에이 인크. 자가환형화 rna의 제조 방법
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
CN121057577A (zh) 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
CN121358494A (zh) 2023-03-15 2026-01-16 旗舰创业创新第六有限责任公司 免疫原性组合物及其用途
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024226621A1 (en) * 2023-04-25 2024-10-31 Ginkgo Bioworks, Inc. Novel internal ribosome entry site (ires) and intron sequences
EP4705481A2 (en) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024254546A2 (en) * 2023-06-08 2024-12-12 Cornell University Virus-like particles comprising circular mrna expression systems and methods of use thereof
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
WO2025034727A1 (en) * 2023-08-07 2025-02-13 Yale University Translational modulating elements for mrna molecules capped with 5'-cap analogs, mrna molecules including the same, and methods using the same
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025072615A1 (en) 2023-09-29 2025-04-03 Orbital Therapeutics, Inc. Improved methods of making rna by splinted ligation and compositions thereof
WO2025077734A1 (en) * 2023-10-09 2025-04-17 Shanghai Circode Biomed Co., Ltd. Constructs and methods for preparing circular rnas and uses thereof
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫受體抑制蛋白及相關方法
WO2025179198A1 (en) 2024-02-23 2025-08-28 Sail Biomedicines, Inc. Circular polyribonucleotides and unmodified linear rnas with reduced immunogenicity
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites
WO2026060273A1 (en) * 2024-09-13 2026-03-19 Shape Therapeutics Inc. 5' untranslated regions (utrs) for enhanced payload expression
WO2026072709A1 (en) 2024-09-25 2026-04-02 Orna Therapeutics, Inc. Chimeric antigen receptors targeting bcma

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236673A1 (en) * 2018-06-06 2019-12-12 Massachusetts Institute Of Technology Circular rna for translation in eukaryotic cells
WO2020023595A1 (en) * 2018-07-24 2020-01-30 Mayo Foundation For Medical Education And Research Circularized engineered rna and methods

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5766903A (en) * 1995-08-23 1998-06-16 University Technology Corporation Circular RNA and uses thereof
US6869779B1 (en) * 1998-08-27 2005-03-22 Fuso Pharmaceutical Industries, Ltd. Nucleic acid sequence for potentiating the expression of useful gene and method therefor
DE10143238A1 (de) * 2001-09-04 2003-03-20 Icon Genetics Ag Identifizierung eukaryotischer interner Ribosomen-Eingangsstellen (IRES)-Elemente
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
US20050118625A1 (en) * 2003-10-02 2005-06-02 Mounts William M. Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases
WO2010084371A1 (en) 2009-01-26 2010-07-29 Mitoprod Novel circular interfering rna molecules
EP2531515B1 (en) * 2010-01-22 2015-10-21 Gen-Probe Incorporated Probes for detecting the presence of trochomonas vaginalis in a sample
DK2925866T3 (en) 2012-11-30 2018-10-29 Univ Aarhus CIRCULAR RNA FOR INHIBITING MICRO-RNA
EP2996697B1 (en) * 2013-05-15 2019-06-26 Robert Kruse Intracellular translation of circular rna
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
EP3374504B1 (en) * 2015-11-09 2025-03-19 CureVac SE Optimized nucleic acid molecules
WO2017222911A1 (en) 2016-06-20 2017-12-28 The Board Of Trustees Of The Leland Stanford Junior University Circular rnas and their use in immunomodulation
JP7385282B2 (ja) * 2017-11-07 2023-11-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 環状rna分子のための方法及び組成物
WO2020023655A1 (en) * 2018-07-24 2020-01-30 Flagship Pioneering, Inc. Compositions comprising circular polyribonucleotides and uses thereof
US20220362295A1 (en) 2019-04-22 2022-11-17 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using fusion proteins

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236673A1 (en) * 2018-06-06 2019-12-12 Massachusetts Institute Of Technology Circular rna for translation in eukaryotic cells
US20200080106A1 (en) * 2018-06-06 2020-03-12 Massachusetts Institute Of Technology Circular rna for translation in eukaryotic cells
WO2020023595A1 (en) * 2018-07-24 2020-01-30 Mayo Foundation For Medical Education And Research Circularized engineered rna and methods

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF MOLECULAR CELL BIOLOGY, vol. 11(10), JPN6025032432, 2019, pages 911 - 919, ISSN: 0005792949 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025520771A (ja) * 2022-06-29 2025-07-03 ソウル大学校産学協力団 RNAの安定性又はmRNA翻訳増強用の新規調節エレメントおよびこれと相互作用するZCCHC2、並びにその用途

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WO2021263124A3 (en) 2022-02-03
US11560567B2 (en) 2023-01-24
CN116323945A (zh) 2023-06-23
US20230125935A1 (en) 2023-04-27
AU2021297340A1 (en) 2023-01-19
US20230407317A1 (en) 2023-12-21
EP4171631A4 (en) 2025-02-12
CA3180224A1 (en) 2021-12-30
US11685924B2 (en) 2023-06-27
WO2021263124A2 (en) 2021-12-30
US20220251578A1 (en) 2022-08-11
KR20230028375A (ko) 2023-02-28
EP4171631A2 (en) 2023-05-03

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