CN113412331A - 闭合端DNA(ceDNA)及其在减少与基因或核酸疗法相关的免疫应答的方法中的用途 - Google Patents

闭合端DNA(ceDNA)及其在减少与基因或核酸疗法相关的免疫应答的方法中的用途 Download PDF

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CN113412331A
CN113412331A CN202080010834.4A CN202080010834A CN113412331A CN 113412331 A CN113412331 A CN 113412331A CN 202080010834 A CN202080010834 A CN 202080010834A CN 113412331 A CN113412331 A CN 113412331A
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inhibitor
vector
itrs
cedna
immune response
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D·A·科尔
P·萨马约亚
R·M·科廷
M·G·斯坦顿
O·阿尔坎
M·齐奥科
R·拉简德兰
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Generational Biology Co
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Generational Biology Co
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CN202080010834.4A 2019-01-24 2020-01-24 闭合端DNA(ceDNA)及其在减少与基因或核酸疗法相关的免疫应答的方法中的用途 Pending CN113412331A (zh)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201962796417P 2019-01-24 2019-01-24
US201962796450P 2019-01-24 2019-01-24
US62/796,417 2019-01-24
US62/796,450 2019-01-24
US201962800303P 2019-02-01 2019-02-01
US201962800285P 2019-02-01 2019-02-01
US62/800,285 2019-02-01
US62/800,303 2019-02-01
US201962814414P 2019-03-06 2019-03-06
US201962814424P 2019-03-06 2019-03-06
US62/814,414 2019-03-06
US62/814,424 2019-03-06
US201962857542P 2019-06-05 2019-06-05
US62/857,542 2019-06-05
PCT/US2020/015026 WO2020154645A1 (en) 2019-01-24 2020-01-24 Close-ended dna (cedna) and use in methods of reducing gene or nucleic acid therapy related immune response

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CN113412331A true CN113412331A (zh) 2021-09-17

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CN202080010834.4A Pending CN113412331A (zh) 2019-01-24 2020-01-24 闭合端DNA(ceDNA)及其在减少与基因或核酸疗法相关的免疫应答的方法中的用途

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US (1) US20220119840A1 (https=)
EP (1) EP3914717A4 (https=)
JP (1) JP2022518504A (https=)
KR (1) KR20210119416A (https=)
CN (1) CN113412331A (https=)
AU (1) AU2020211457A1 (https=)
CA (1) CA3127799A1 (https=)
MA (1) MA54826A (https=)
MX (1) MX2021008874A (https=)
SG (1) SG11202107922QA (https=)
WO (1) WO2020154645A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114796216A (zh) * 2022-01-04 2022-07-29 南京医科大学 甲氟喹在防治全身代谢性炎症疾病中的应用

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
SG11202005271TA (en) * 2018-01-19 2020-07-29 Generation Bio Co Closed-ended dna vectors obtainable from cell-free synthesis and process for obtaining cedna vectors
CN108920903B (zh) * 2018-07-09 2022-04-01 湘潭大学 基于朴素贝叶斯的LncRNA与疾病的关联关系预测方法及系统
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3956448A4 (en) * 2019-04-18 2022-10-19 University of Massachusetts AIM2 INHIBITORS AND USES THEREOF
US20210371465A1 (en) * 2020-03-26 2021-12-02 Yale University Method of using a tlr9 antagonist as an anti-inflammatory and anti-fibrotic agent
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
US20220090130A1 (en) * 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CN112138164B (zh) * 2020-09-25 2022-06-03 徐州医科大学 Aim2在制备降低car-t治疗毒副作用的药物中的应用
EP4297728A1 (en) 2021-02-23 2024-01-03 Poseida Therapeutics, Inc. Compositions and methods for delivery of nucleic acids
CA3230386A1 (en) * 2021-07-23 2023-01-26 Spark Therapeutics, Inc. Methods and composition for non-viral dna delivery
AU2022211795A1 (en) * 2021-08-06 2023-02-23 Syte.Bio Inc. Hairpin loop ended self-complementary double-stranded covalently closed linear dna vector, manufacturing system and process, and uses of said resulting dna vector
JP2024532476A (ja) * 2021-09-02 2024-09-05 モレキュラー アクシオム エルエルシー Ｎｌｒｐ３またはｎｌｒｐ１発現を調節するための組成物及び方法
JP2025532680A (ja) * 2022-09-20 2025-10-01 ザ・カウンシル・オヴ・ザ・クイーンズランド・インスティテュート・オヴ・メディカル・リサーチ 抗線維化マイクロｒｎａ組成物
EP4630561A1 (en) 2022-12-09 2025-10-15 Takeda Pharmaceutical Company Limited Modified immunomodulators
AU2024224841A1 (en) 2023-02-21 2025-08-21 Poseida Therapeutics, Inc. Aav piggybac transposon polynucleotide compositions and methods of use therefor
CN121532216A (zh) * 2023-04-18 2026-02-13 赛仁生物技术公司 腺相关病毒载体组合物和使用方法
WO2025064507A1 (en) 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140242093A1 (en) * 2011-07-29 2014-08-28 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
WO2017214378A1 (en) * 2016-06-08 2017-12-14 President And Fellows Of Harvard College Engineered viral vector reduces induction of inflammatory and immune responses
CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8377898B2 (en) * 2006-10-12 2013-02-19 Idera Pharmaceuticals, Inc. Immune regulatory oligonucleotide (IRO) compounds to modulate toll-like receptor based immune response
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
ES2977259T3 (es) * 2014-11-05 2024-08-21 Cartesian Therapeutics Inc Métodos y composiciones relacionadas con nanovehículos sintéticos con rapamicina en un estado sobresaturado estable

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140242093A1 (en) * 2011-07-29 2014-08-28 Fred Hutchinson Cancer Research Center Methods and compositions for modulating the innate immune response and/or myogenesis in a mammalian subject
CN109195636A (zh) * 2016-03-03 2019-01-11 马萨诸塞大学 用于非病毒基因转移的末端封闭型线性双链体dna
WO2017214378A1 (en) * 2016-06-08 2017-12-14 President And Fellows Of Harvard College Engineered viral vector reduces induction of inflammatory and immune responses

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
KARL MARAMOROSCH等编著: "《Advances in Virus Research》", ACADEMIC PRESS, pages: 63 - 100 *
VINCENT J等: "Small molecule inhibition of cGAS reduces interferon expression in primary macrophages from autoimmune mice.", 《NAT COMMUN》, pages 1827 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114796216A (zh) * 2022-01-04 2022-07-29 南京医科大学 甲氟喹在防治全身代谢性炎症疾病中的应用

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