TW202317767A - 環狀rna組合物及方法 - Google Patents

環狀rna組合物及方法 Download PDF

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Publication number
TW202317767A
TW202317767A TW111121745A TW111121745A TW202317767A TW 202317767 A TW202317767 A TW 202317767A TW 111121745 A TW111121745 A TW 111121745A TW 111121745 A TW111121745 A TW 111121745A TW 202317767 A TW202317767 A TW 202317767A
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Taiwan
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sequence
rna polynucleotide
precursor rna
polynucleotide according
fragment
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TW111121745A
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Chinese (zh)
Inventor
羅伯特 亞歷山大 魏索霍夫特
克莉絲汀 奧特
托馬斯 巴恩斯
格雷戈里 莫茲
艾咪 Ｍ 貝克
艾倫 Ｔ 霍哈塔
布萊恩 古德曼
舒緩
瓦倫 席凡夏卡
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美商歐納醫療公司
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Publication of TW202317767A publication Critical patent/TW202317767A/zh

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TW111121745A 2021-06-10 2022-06-10 環狀rna組合物及方法 TW202317767A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163209271P 2021-06-10 2021-06-10
US63/209,271 2021-06-10
US202263311923P 2022-02-18 2022-02-18
US63/311,923 2022-02-18

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TW202317767A true TW202317767A (zh) 2023-05-01

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US (1) US20250283073A1 (OSRAM)
EP (1) EP4352234A2 (OSRAM)
JP (1) JP2024521444A (OSRAM)
KR (1) KR20240019791A (OSRAM)
AU (1) AU2022288705A1 (OSRAM)
BR (1) BR112023025515A2 (OSRAM)
CA (1) CA3219932A1 (OSRAM)
IL (1) IL308746A (OSRAM)
MX (1) MX2023014702A (OSRAM)
TW (1) TW202317767A (OSRAM)
WO (1) WO2022261490A2 (OSRAM)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK4271818T3 (da) 2021-09-17 2024-11-04 Flagship Pioneering Innovations Vi Llc Sammensætninger og fremgangsmåder til fremstilling af cirkulære polyribonukleotider
CN114214237B (zh) * 2021-12-23 2023-06-16 南京大学 一种洗脱细胞表面吸附纳米颗粒的方法
TW202334412A (zh) * 2022-01-11 2023-09-01 北京大學 針對 sars-cov-2 變體的環狀 rna 疫苗及其使用方法
CA3241143A1 (en) * 2022-01-21 2023-07-27 Allen T. HORHOTA Systemic administration of circular rna polynucleotides encoding muscle proteins or protein complexes
WO2023182948A1 (en) * 2022-03-21 2023-09-28 Bio Adventure Co., Ltd. Internal ribosome entry site (ires), plasmid vector and circular mrna for enhancing protein expression
US12297285B2 (en) * 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN116064531B (zh) * 2022-09-07 2024-04-02 昆明理工大学 一种抑制cvb5病毒复制的长链非编码rna及应用
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
EP4634390A2 (en) 2022-12-15 2025-10-22 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
KR20240159310A (ko) * 2023-04-28 2024-11-05 주식회사 메디치바이오 신규한 이온화지질을 이용한 지질나노입자 조성물
WO2024205657A2 (en) * 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
JP2026512425A (ja) 2023-03-30 2026-04-16 オーエスイー・イミュノセラピューティクス 免疫細胞増強分子の発現のための活性化免疫細胞に標的化された脂質ベースのナノ粒子及びその使用
EP4687990A1 (en) 2023-03-30 2026-02-11 Ose Immunotherapeutics Method of synthesis of targeted lipid nanoparticle and uses thereof
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
EP4698150A1 (en) 2023-04-19 2026-02-25 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
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025006613A1 (en) * 2023-06-27 2025-01-02 Hopewell Therapeutics, Inc. Lipid nanoparticles for cancer immunotherapy
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
WO2025036467A1 (en) * 2023-08-17 2025-02-20 Wuhan University Circular rna with increased stability and expression
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
WO2025117969A1 (en) 2023-12-01 2025-06-05 Orna Therapeutics, Inc. Process for manufacturing lipid nanoparticles
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025133115A1 (en) 2023-12-21 2025-06-26 Ose Immunotherapeutics Lipid-based nanoparticles comprising il-35
WO2025149040A1 (zh) * 2024-01-11 2025-07-17 深圳近邻生物科技有限公司 一种通用的且无"scar"的RNA环化方法
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025179198A1 (en) 2024-02-23 2025-08-28 Sail Biomedicines, Inc. Circular polyribonucleotides and unmodified linear rnas with reduced immunogenicity
WO2025218812A1 (en) * 2024-04-19 2025-10-23 Shanghai Circode Biomed Co., Ltd. Novel internal ribosome entry sites and uses thereof
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
CN118703488A (zh) * 2024-06-20 2024-09-27 毕昇(北京)生物科技有限公司 一种环状rna的纯化方法
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites
WO2026072709A1 (en) 2024-09-25 2026-04-02 Orna Therapeutics, Inc. Chimeric antigen receptors targeting bcma
WO2026068705A1 (en) 2024-09-26 2026-04-02 Ose Immunotherapeutics Lipid-based nanoparticles comprising non-glycosylated fc domains and uses thereof
GB202415784D0 (en) * 2024-10-25 2024-12-11 Rnavate Ltd Methods for purifying circular ribonucleic acid (RNA)
WO2026093414A1 (en) 2024-10-30 2026-05-07 Ose Immunotherapeutics Lipid-based nanoparticles comprising membrane il-35
WO2026096738A1 (en) 2024-10-31 2026-05-07 Orna Therapeutics, Inc. Chimeric antigen receptors targeting cd19
US20260115324A1 (en) * 2024-10-31 2026-04-30 Orna Therapeutics, Inc. Chimeric Antigen Receptors Targeting CD19
CN119876152A (zh) * 2025-02-10 2025-04-25 毕昇(北京)生物科技有限公司 一种环状rna、嵌合抗原受体t细胞和构建方法及应用

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
IL104570A0 (en) 1992-03-18 1993-05-13 Yeda Res & Dev Chimeric genes and cells transformed therewith
US6066626A (en) 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method for treating lysosomal storage disease
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
WO2001060414A2 (en) 2000-02-17 2001-08-23 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
MXPA05000511A (es) 2001-07-12 2005-09-30 Jefferson Foote Anticuepros super humanizados.
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
JP5563194B2 (ja) 2004-06-29 2014-07-30 イムノコア リミテッド 改変ｔ細胞レセプターを発現する細胞
PT3083556T (pt) 2013-12-19 2020-03-05 Novartis Ag Lípidos e composições lipídicas para a entrega de agentes ativos
RS63030B1 (sr) 2015-09-17 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
US11814627B2 (en) * 2016-06-20 2023-11-14 The Board Of The Leland Stanford Junior University Circular RNAs and their use in immunomodulation
WO2019081383A1 (en) 2017-10-25 2019-05-02 Universität Zürich EUCARYOTE 4 INITIATION FACTOR RECRUITMENT APTAMERS FOR ENHANCED TRANSLATION
US20190314291A1 (en) 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
BR112020015796A2 (pt) 2018-02-01 2021-02-09 Trustees Of Tufts College nanocomplexos semelhantes a lipídios e usos dos mesmos
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
KR20210018323A (ko) * 2018-06-06 2021-02-17 매사추세츠 인스티튜트 오브 테크놀로지 진핵 세포에서의 번역을 위한 원형 rna
JP7686574B2 (ja) * 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状ｒｎａ組成物及び方法

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