MX2022011677A - Composiciones y métodos de arn circular. - Google Patents

Composiciones y métodos de arn circular.

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Publication number
MX2022011677A
MX2022011677A MX2022011677A MX2022011677A MX2022011677A MX 2022011677 A MX2022011677 A MX 2022011677A MX 2022011677 A MX2022011677 A MX 2022011677A MX 2022011677 A MX2022011677 A MX 2022011677A MX 2022011677 A MX2022011677 A MX 2022011677A
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Mexico
Prior art keywords
circular rna
methods
rna
expression
compared
Prior art date
Application number
MX2022011677A
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English (en)
Inventor
Brian Goodman
Alex Wesselhoeft
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Orna Therapeutics Inc
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Publication date
Application filed by Orna Therapeutics Inc filed Critical Orna Therapeutics Inc
Publication of MX2022011677A publication Critical patent/MX2022011677A/es

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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
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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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07K14/4748Tumour specific antigens; Tumour rejection antigen precursors [TRAP], e.g. MAGE
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    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
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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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    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/60Vectors comprising a special translation-regulating system from viruses

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  • Animal Behavior & Ethology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
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Abstract

En la presente se describe ARN circular, junto con composiciones y métodos relacionados. En algunas modalidades, el ARN circular inventivo comprende un grupo I de posempalme en fragmentos de hierro, espaciadores, un IRES, regiones formadoras de dobles opcionales y más de una secuencia de expresión. En algunas modalidades, las secuencias de expresión se encuentran separadas por una o más secuencias de polinucleótidos que codifican un sitio de escisión. En algunas modalidades, el ARN circular de la invención tiene mejor expresión, estabilidad funcional, inmunogenicidad, facilidad de elaboración y/o semivida en comparación con el ARN lineal. En algunas modalidades, los métodos y construcciones inventivos producen una mejor eficiencia de circularización, eficiencia de empalme y/o pureza en comparación con los enfoques existentes de circularización de ARN.
MX2022011677A 2020-03-20 2021-03-22 Composiciones y métodos de arn circular. MX2022011677A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202062992518P 2020-03-20 2020-03-20
PCT/US2021/023540 WO2021189059A2 (en) 2020-03-20 2021-03-22 Circular rna compositions and methods

Publications (1)

Publication Number Publication Date
MX2022011677A true MX2022011677A (es) 2023-01-11

Family

ID=75539941

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022011677A MX2022011677A (es) 2020-03-20 2021-03-22 Composiciones y métodos de arn circular.

Country Status (9)

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EP (1) EP4121453A2 (es)
JP (1) JP2023518295A (es)
KR (1) KR20230069042A (es)
CN (1) CN116034114A (es)
AU (1) AU2021237738A1 (es)
BR (1) BR112022018854A2 (es)
CA (1) CA3172423A1 (es)
MX (1) MX2022011677A (es)
WO (1) WO2021189059A2 (es)

Families Citing this family (16)

* 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
WO2022191642A1 (ko) 2021-03-10 2022-09-15 알지노믹스 주식회사 자가 환형화 rna 구조체
CN113999852B (zh) * 2021-11-30 2024-04-02 杭州市富阳区第一人民医院 circ_0001772作为结直肠癌诊断和治疗标志物的应用
WO2023107593A2 (en) * 2021-12-09 2023-06-15 Carisma Therapeutics Inc. In vivo delivery to immune cells
CN114574483B (zh) * 2022-03-02 2024-05-10 苏州科锐迈德生物医药科技有限公司 基于翻译起始元件点突变的重组核酸分子及其在制备环状rna中的应用
CN114438127B (zh) * 2022-03-02 2024-03-19 苏州科锐迈德生物医药科技有限公司 一种重组核酸分子及其在制备环状rna中的应用
AU2023240827A1 (en) * 2022-03-21 2024-07-18 Bio Adventure Co., Ltd. Internal ribosome entry site (ires), plasmid vector and circular mrna for enhancing protein expression
WO2024008189A1 (en) * 2022-07-08 2024-01-11 Shanghai Circode Biomed Co., Ltd. Methods and systems for purifying circular nucleic acids
WO2024030957A1 (en) * 2022-08-02 2024-02-08 William Marsh Rice University Compositions and methods for facilitating heart and lung repair
WO2024092001A1 (en) * 2022-10-26 2024-05-02 Memorial Sloan-Kettering Cancer Center Cd33 antibody compositions for treating alzheimer's disease
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
WO2024112717A1 (en) * 2022-11-21 2024-05-30 Avstera Therapeutics Corp. Mrna composition for preventing and treating cancer
WO2024129982A2 (en) 2022-12-15 2024-06-20 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
CN116284006B (zh) * 2023-05-10 2023-08-25 北京因诺惟康医药科技有限公司 可电离脂质化合物、包含其的脂质载体及应用
CN118222638A (zh) * 2024-05-22 2024-06-21 中国农业大学 一种干扰蚯蚓目的基因表达的方法

Family Cites Families (26)

* 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
ATE333893T1 (de) 1997-10-29 2006-08-15 Genzyme Corp Gentherapie für gaucher-krankheit
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
JP2005538706A (ja) 2001-07-12 2005-12-22 ジェファーソン フーテ, スーパーヒト化抗体
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
BR0316880A (pt) 2002-12-23 2005-10-25 Wyeth Corp Anticorpos contra pd-1 e usos dos mesmos
ATE475669T1 (de) 2004-06-29 2010-08-15 Immunocore Ltd Einen modifizierten t-zellen-rezeptor exprimierende zellen
PT2439273T (pt) 2005-05-09 2019-05-13 Ono Pharmaceutical Co Anticorpos monoclonais humanos para morte programada 1 (pd- 1) e métodos para o tratamento de cancro utilizando anticorpos anti-pd-1 isoladamente ou em combinação com outros imunoterapêuticos
US8747847B2 (en) 2008-02-11 2014-06-10 Curetech Ltd. Monoclonal antibodies for tumor treatment
EP2262837A4 (en) 2008-03-12 2011-04-06 Merck Sharp & Dohme PD-1 BINDING PROTEINS
CN104740610A (zh) 2008-08-25 2015-07-01 安普利穆尼股份有限公司 Pd-1拮抗剂及其使用方法
EP2350129B1 (en) 2008-08-25 2015-06-10 Amplimmune, Inc. Compositions of pd-1 antagonists and methods of use
EP2504028A4 (en) 2009-11-24 2014-04-09 Amplimmune Inc SIMULTANEOUS INHIBITION OF PD-L1 / PD-L2
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
RS63030B1 (sr) 2015-09-17 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava
HUE061564T2 (hu) 2015-10-28 2023-07-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske készítmények nukleinsavak bevitelére
BR112020011670A2 (pt) * 2017-12-15 2020-11-17 Flagship Pioneering Innovations Vi, Llc. composições compreendendo polirribonucleotídeos circulares e seus usos
EP3746052A1 (en) * 2018-01-30 2020-12-09 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
KR20200115591A (ko) 2018-02-01 2020-10-07 트러스티즈 오브 터프츠 칼리지 지질-유사 나노복합체 및 이의 용도
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
CA3104034A1 (en) * 2018-05-01 2019-11-07 Fred Hutchinson Cancer Research Center Nanoparticles for gene expression and uses thereof
WO2019222275A2 (en) * 2018-05-14 2019-11-21 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using inducible fusion proteins
EP3826643A4 (en) * 2018-07-24 2022-05-18 Mayo Foundation for Medical Education and Research CIRCULARIZED MANIPULATED RNA AND METHODS
CN115867291A (zh) * 2019-05-22 2023-03-28 麻省理工学院 环状rna组合物和方法

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Publication number Publication date
CN116034114A (zh) 2023-04-28
AU2021237738A1 (en) 2022-11-10
EP4121453A2 (en) 2023-01-25
JP2023518295A (ja) 2023-04-28
CA3172423A1 (en) 2021-03-22
WO2021189059A3 (en) 2021-11-11
KR20230069042A (ko) 2023-05-18
BR112022018854A2 (pt) 2023-03-07
WO2021189059A2 (en) 2021-09-23

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