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HK1220122A1 - Mrna的异源未转录区域 - Google Patents

Mrna的异源未转录区域 Download PDF

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Publication number
HK1220122A1
HK1220122A1 HK16108198.5A HK16108198A HK1220122A1 HK 1220122 A1 HK1220122 A1 HK 1220122A1 HK 16108198 A HK16108198 A HK 16108198A HK 1220122 A1 HK1220122 A1 HK 1220122A1
Authority
HK
Hong Kong
Prior art keywords
mrna
untranslated regions
heterologous untranslated
heterologous
optimization
Prior art date
Application number
HK16108198.5A
Other languages
English (en)
Inventor
Tirtha Chakraborty
Antonin De Fougerolles
Original Assignee
Modernatx, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Modernatx, Inc. filed Critical Modernatx, Inc.
Publication of HK1220122A1 publication Critical patent/HK1220122A1/zh

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/193Colony stimulating factors [CSF]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
HK16108198.5A 2013-03-09 2014-03-07 Mrna的异源未转录区域 HK1220122A1 (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361775509P 2013-03-09 2013-03-09
US201361775509P 2013-03-09
US201361829372P 2013-05-31 2013-05-31
US201361829372P 2013-05-31
PCT/US2014/021522 WO2014164253A1 (en) 2013-03-09 2014-03-07 Heterologous untranslated regions for mrna

Publications (1)

Publication Number Publication Date
HK1220122A1 true HK1220122A1 (zh) 2017-04-28

Family

ID=51658835

Family Applications (1)

Application Number Title Priority Date Filing Date
HK16108198.5A HK1220122A1 (zh) 2013-03-09 2014-03-07 Mrna的异源未转录区域

Country Status (4)

Country Link
US (2) US20160022840A1 (zh)
EP (1) EP2964234A4 (zh)
HK (1) HK1220122A1 (zh)
WO (1) WO2014164253A1 (zh)

Families Citing this family (202)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN104531812A (zh) 2010-10-01 2015-04-22 现代治疗公司 设计核酸及其使用方法
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
RU2670745C9 (ru) 2011-05-24 2018-12-13 Бионтех Рна Фармасьютикалс Гмбх Индивидуализированные противоопухолевые вакцины
PL2791160T3 (pl) 2011-12-16 2022-06-20 Modernatx, Inc. Kompozycje zmodyfikowanego mrna
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
HK1206779A1 (zh) 2012-04-02 2016-01-15 Modernatx, Inc. 生产核蛋白的改性的多核苷酸
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
EP2925348B1 (en) 2012-11-28 2019-03-06 BioNTech RNA Pharmaceuticals GmbH Individualized vaccines for cancer
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
US10138507B2 (en) 2013-03-15 2018-11-27 Modernatx, Inc. Manufacturing methods for production of RNA transcripts
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3157573A4 (en) 2014-06-19 2018-02-21 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
EP3169335B8 (en) 2014-07-16 2019-10-09 ModernaTX, Inc. Circular polynucleotides
AU2015289573A1 (en) * 2014-07-17 2017-02-02 Modernatx, Inc Terminal modifications of polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
WO2016100812A1 (en) * 2014-12-19 2016-06-23 Moderna Therapeutics, Inc. Terminal modifications of polynucleotides
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
AU2016316439B2 (en) 2015-08-28 2022-02-24 CureVac SE Artificial nucleic acid molecules
AU2016324463B2 (en) 2015-09-17 2022-10-27 Modernatx, Inc. Polynucleotides containing a stabilizing tail region
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
ES2810701T5 (es) 2015-10-05 2024-07-11 Modernatx Inc Procedimientos para la administración terapéutica de medicamentos de ácido ribonucleico mensajero
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
US20180312545A1 (en) 2015-11-09 2018-11-01 Curevac Ag Optimized nucleic acid molecules
WO2017100551A1 (en) * 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
SI3394093T1 (sl) 2015-12-23 2022-05-31 Modernatx, Inc. Metode uporabe liganda OX40, ki kodira polinukleotid
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
EP3405579A1 (en) 2016-01-22 2018-11-28 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
US20190167811A1 (en) 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
JP7114485B2 (ja) 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα-ガラクトシダーゼAをコードするポリヌクレオチド
WO2017201332A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency
EP3458105B1 (en) 2016-05-18 2024-01-17 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP7210287B2 (ja) 2016-05-18 2023-01-23 モダーナティエックス・インコーポレイテッド Ii型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド
JP7088911B2 (ja) 2016-05-18 2022-06-21 モデルナティエックス インコーポレイテッド リラキシンをコードするポリヌクレオチド
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
CA3024624A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
EP4137509A1 (en) 2016-05-18 2023-02-22 ModernaTX, Inc. Combinations of mrnas encoding immune modulating polypeptides and uses thereof
SG11201809381XA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding interleukin-12 (il12) and uses thereof
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
MX2019000205A (es) 2016-07-07 2019-09-23 Rubius Therapeutics Inc Composiciones y metodos relacionados con sistemas celulares terapeuticos que expresan arn exogeno.
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
CA3042015A1 (en) 2016-10-26 2018-05-03 Modernatx, Inc. Messenger ribonucleic acids for enhancing immune responses and methods of use thereof
CN110325210A (zh) 2017-01-11 2019-10-11 小利兰·斯坦福大学托管委员会 Rspo替代物分子
WO2018140821A1 (en) 2017-01-26 2018-08-02 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
SG11201906969PA (en) 2017-02-01 2019-08-27 Modernatx Inc Immunomodulatory therapeutic mrna compositions encoding activating oncogene mutation peptides
US11421011B2 (en) 2017-05-18 2022-08-23 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (IL12) polypeptides and uses thereof
WO2018213789A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Modified messenger rna comprising functional rna elements
CA3063907A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
WO2018224166A1 (en) 2017-06-09 2018-12-13 Biontech Rna Pharmaceuticals Gmbh Methods for predicting the usefulness of disease specific amino acid modifications for immunotherapy
US20200268666A1 (en) 2017-06-14 2020-08-27 Modernatx, Inc. Polynucleotides encoding coagulation factor viii
WO2018231990A2 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3668522B1 (en) 2017-08-18 2024-09-11 ModernaTX, Inc. Efficacious mrna vaccines
EP3714045A1 (en) 2017-11-22 2020-09-30 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
JP7423522B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
IL275205B2 (en) 2017-12-15 2025-03-01 Flagship Pioneering Innovations Vi Llc Preparations containing circular polyribonucleotides and uses thereof
MA51523A (fr) 2018-01-05 2020-11-11 Modernatx Inc Polynucléotides codant pour des anticorps anti-virus du chikungunya
EP3737424A4 (en) 2018-01-10 2021-10-27 Translate Bio MA, Inc. COMPOSITIONS AND METHODS TO FACILITATE THE DELIVERY OF SYNTHETIC NUCLEIC ACIDS INTO CELLS
KR102141124B1 (ko) 2018-01-30 2020-08-04 (주)바이오니아 이중 가닥 miRNA를 포함하는 이중나선 올리고뉴클레오타이드 구조체 및 이의 용도
AU2019216307A1 (en) 2018-01-30 2020-07-09 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
WO2019195765A1 (en) * 2018-04-06 2019-10-10 University Of Massachusetts Mlk-regulated micrornas in angiogenesis and tumor development
US20210163928A1 (en) 2018-04-11 2021-06-03 Modernatx, Inc. Messenger rna comprising functional rna elements
CN119286871A (zh) 2018-04-19 2025-01-10 查美特制药公司 合成rig-i样受体激动剂
WO2020014271A1 (en) 2018-07-09 2020-01-16 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020047201A1 (en) 2018-09-02 2020-03-05 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
CN108977533B (zh) * 2018-09-10 2021-09-17 镇江市第三人民医院 一种用于预测慢性乙肝炎症损伤的miRNA组合物
CN109022569B (zh) * 2018-09-10 2021-09-17 镇江市第三人民医院 一种用于预测慢性乙肝肝纤维化的miRNA组合物
MX2021003015A (es) 2018-09-13 2021-07-15 Modernatx Inc Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.
MA53608A (fr) 2018-09-13 2021-07-21 Modernatx Inc Polynucléotides codant pour les sous-unités e1-alpha, e1-beta et e2 du complexe alpha-cétoacide déshydrogénase à chaîne ramifiée pour le traitement de la leucinose
US20230081530A1 (en) 2018-09-14 2023-03-16 Modernatx, Inc. Methods and compositions for treating cancer using mrna therapeutics
EP3850102A1 (en) 2018-09-14 2021-07-21 ModernaTX, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
CN109055541B (zh) * 2018-09-26 2020-08-28 上海市精神卫生中心(上海市心理咨询培训中心) Ad所致mci诊断标志物及其应用
US20220152225A1 (en) 2018-09-27 2022-05-19 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
US20220001026A1 (en) 2018-11-08 2022-01-06 Modernatx, Inc. Use of mrna encoding ox40l to treat cancer in human patients
HUE064076T2 (hu) 2018-12-06 2024-02-28 Arcturus Therapeutics Inc Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezelésére
WO2020198403A2 (en) 2019-03-25 2020-10-01 Flagship Pioneering Innovations Vi, Llc Compositions comprising modified circular polyribonucleotides and uses thereof
EP3966333A1 (en) 2019-05-07 2022-03-16 ModernaTX, Inc. Polynucleotides for disrupting immune cell activity and methods of use thereof
MA55887A (fr) 2019-05-07 2022-03-16 Modernatx Inc Microarn de cellules immunitaires exprimés de manière différentielle pour la régulation de l'expression de protéines
EP3965806A1 (en) 2019-05-08 2022-03-16 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
US20220226438A1 (en) 2019-05-08 2022-07-21 Astrazeneca Ab Compositions for skin and wounds and methods of use thereof
MA56517A (fr) 2019-06-24 2022-04-27 Modernatx Inc Arn messager comprenant des éléments d'arn fonctionnels et leurs utilisations
MA56539A (fr) 2019-06-24 2022-04-27 Modernatx Inc Arn messager résistant à l'endonucléase et utilisations correspondantes
CA3145446A1 (en) 2019-07-02 2021-01-07 Fundacion Para La Investigacion Medica Aplicada Cpla2e inducing agents and uses thereof
WO2021050986A1 (en) 2019-09-11 2021-03-18 Modernatx, Inc. Lnp-formulated mrna therapeutics and use thereof for treating human subjects
AU2020366209A1 (en) 2019-10-15 2022-05-19 Board Of Regents Of The University Of Nebraska mRNA encoding granulocyte-macrophage colony stimulating factor for treating Parkinson's disease
US20220378817A1 (en) 2019-10-23 2022-12-01 Checkmate Pharmaceuticals, Inc. Synthetic rig-i-like receptor agonists
CA3165956A1 (en) * 2019-12-26 2021-07-01 National University Corporation Tokai National Higher Education And Research System Polynucleotide and pharmaceutical composition
CA3169889A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
CN114875140B (zh) * 2020-03-30 2025-07-04 中国医学科学院肿瘤医院 包括外泌体miR-106b-3p、miR-1294等在肺癌诊断中的应用
CN114214423B (zh) * 2020-03-30 2023-06-27 中国医学科学院肿瘤医院 外泌体miR-30e-5p、IL1B等在肺癌诊断中的应用
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
WO2021231854A1 (en) 2020-05-14 2021-11-18 Modernatx, Inc. Lnp compositions comprising an mrna therapeutic and an effector molecule
EP4157319A1 (en) 2020-05-28 2023-04-05 Modernatx, Inc. Use of mrnas encoding ox40l, il-23 and il-36gamma for treating cancer
JP2023527875A (ja) 2020-06-01 2023-06-30 モダーナティエックス・インコーポレイテッド フェニルアラニンヒドロキシラーゼバリアント及びその使用
EP4157217A1 (en) 2020-06-01 2023-04-05 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
CN116194151A (zh) 2020-06-23 2023-05-30 摩登纳特斯有限公司 包含具有延长的半衰期的mRNA治疗剂的LNP组合物
AU2021320426A1 (en) 2020-08-06 2023-03-23 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
AU2021379770A1 (en) 2020-11-16 2023-06-22 Surrozen Operating, Inc. Liver-specific wnt signal enhancing molecules and uses thereof
CN114717229B (zh) * 2021-01-05 2024-09-10 麦塞拿治疗(香港)有限公司 治疗性mRNA的无细胞和无载体体外RNA转录方法和核酸分子
US20240148794A1 (en) 2021-02-12 2024-05-09 Modernatx, Inc. Lnp compositions comprising payloads for in vivo therapy
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
JP2024512026A (ja) 2021-03-24 2024-03-18 モデルナティエックス インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療を目的とした脂質ナノ粒子及びオルニチントランスカルバミラーゼをコードするポリヌクレオチド
US20240207444A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
US20240229109A1 (en) 2021-04-01 2024-07-11 Modernatx, Inc. Methods for identification and ratio determination of rna species in multivalent rna compositions
US20240269248A1 (en) 2021-05-19 2024-08-15 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
KR20240017865A (ko) 2021-06-04 2024-02-08 트랜슬레이트 바이오 인코포레이티드 Mrna 캡핑 효율의 정량적 평가를 위한 분석
US20240384277A1 (en) 2021-06-15 2024-11-21 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
US20240218353A1 (en) 2021-06-17 2024-07-04 Modernatx, Inc. Alternative rna purification strategies
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
JP2024528447A (ja) * 2021-06-24 2024-07-30 ハンミ ファーマシューティカルズ カンパニー リミテッド 非天然型5’非翻訳領域及び3’非翻訳領域、及びその用途
US20240316165A1 (en) 2021-07-12 2024-09-26 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
WO2023009422A1 (en) 2021-07-26 2023-02-02 Modernatx, Inc. Processes for preparing lipid nanoparticle compositions for the delivery of payload molecules to airway epithelium
TW202320736A (zh) 2021-07-26 2023-06-01 美商現代公司 用於製備脂質奈米粒子組成物之製程
US20250090684A1 (en) 2021-07-27 2025-03-20 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease type 1a (gsd1a)
EP4384613A1 (en) 2021-08-13 2024-06-19 ModernaTX, Inc. Multicolumn chromatography mrna purification
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
US20250017867A1 (en) 2021-10-01 2025-01-16 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
WO2023064469A1 (en) 2021-10-13 2023-04-20 Modernatx, Inc. Compositions of mrna-encoded il15 fusion proteins and methods of use thereof
JP2024539512A (ja) 2021-10-22 2024-10-28 セイル バイオメディシンズ インコーポレイテッド Mrnaワクチン組成物
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023076658A1 (en) 2021-11-01 2023-05-04 Modernatx, Inc. Mass spectrometry of mrna
JP2024540170A (ja) 2021-11-01 2024-10-31 モデルナティエックス インコーポレイテッド インテグリンベータ-6をコードするポリヌクレオチド及びその使用方法
EP4426855A1 (en) 2021-11-05 2024-09-11 ModernaTX, Inc. Methods of purifying dna for gene synthesis
WO2023086465A1 (en) 2021-11-12 2023-05-19 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
CN118488834A (zh) 2021-11-23 2024-08-13 赛欧生物医药股份有限公司 细菌源性脂质组合物和其用途
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
JP2025500373A (ja) 2021-12-20 2025-01-09 セイル バイオメディシンズ インコーポレイテッド Mrna治療用組成物
US20250084397A1 (en) 2022-01-04 2025-03-13 Modernatx, Inc. Methods of purifying dna for gene synthesis
WO2023137149A1 (en) 2022-01-14 2023-07-20 Modernatx, Inc. In vitro transcription dna purification and recycling
EP4469091A1 (en) 2022-01-28 2024-12-04 CureVac SE Nucleic acid encoded transcription factor inhibitors
EP4473107A1 (en) 2022-02-03 2024-12-11 ModernaTX, Inc. Continuous precipitation for mrna purification
US20250235531A1 (en) 2022-02-09 2025-07-24 Modernatx, Inc. Mucosal administration methods and formulations
TW202345864A (zh) 2022-02-18 2023-12-01 美商現代公司 編碼檢查點癌症疫苗之mRNA及其用途
CN114652738B (zh) * 2022-03-04 2022-11-18 中国医学科学院北京协和医院 miR-1285-5p在薄型子宫内膜中的应用
WO2023196399A1 (en) 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023196988A1 (en) 2022-04-07 2023-10-12 Modernatx, Inc. Methods of use of mrnas encoding il-12
EP4508242A1 (en) 2022-04-11 2025-02-19 ModernaTX, Inc. Detection of mrna purity in a mixture
US20250251391A1 (en) 2022-04-15 2025-08-07 Modernatx, Inc. Ribosomal engagement potency assay
KR20250004778A (ko) 2022-04-15 2025-01-08 스마트셀라 솔루션즈 아베 mRNA 제제의 엑소좀-매개 전달을 위한 조성물 및 방법
WO2023215498A2 (en) 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
WO2023225524A1 (en) 2022-05-17 2023-11-23 Modernatx, Inc. Preparation of highly concentrated mrna
JP2025517508A (ja) 2022-05-25 2025-06-05 キュアバック エスイー 核酸ベースのワクチン
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
WO2024010993A1 (en) 2022-07-06 2024-01-11 Modernatx, Inc. Primer design for cell-free dna production
WO2024025815A1 (en) 2022-07-25 2024-02-01 Modernatx, Inc. Use of imac to improve rna purity
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
WO2024097874A1 (en) 2022-11-03 2024-05-10 Modernatx, Inc. Chemical stability of mrna
EP4615424A1 (en) 2022-11-10 2025-09-17 Sail Biomedicines, Inc. Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs
WO2024107827A1 (en) 2022-11-16 2024-05-23 The Broad Institute, Inc. Therapeutic exploitation of sting channel activity
WO2024130158A1 (en) 2022-12-16 2024-06-20 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding extended serum half-life interleukin-22 for the treatment of metabolic disease
EP4648793A1 (en) 2023-01-11 2025-11-19 ModernaTX, Inc. Personalized cancer vaccines
AU2024212425A1 (en) 2023-01-27 2025-08-07 Sail Biomedicines, Inc. A modified lipid composition and uses thereof
WO2024178305A1 (en) 2023-02-24 2024-08-29 Modernatx, Inc. Compositions of mrna-encoded il-15 fusion proteins and methods of use thereof for treating cancer
WO2024182301A2 (en) 2023-02-27 2024-09-06 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding galactose-1-phosphate uridylyltransferase (galt) for the treatment of galactosemia
DE112024001143T5 (de) 2023-03-08 2025-12-18 CureVac SE Neue lipid-nanopartikel-formeln für die abgabe von nukleinsäuren
EP4680749A1 (en) 2023-03-15 2026-01-21 Kyoto Prefectural Public University Corporation Peptide expression constructs and uses thereof
WO2024197033A1 (en) 2023-03-21 2024-09-26 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of heart failure
EP4683667A1 (en) 2023-03-23 2026-01-28 ModernaTX, Inc. Peg targeting compounds for delivery of therapeutics
WO2024197307A1 (en) 2023-03-23 2024-09-26 Modernatx, Inc. Peg targeting compounds for delivery of therapeutics
WO2024197309A1 (en) 2023-03-23 2024-09-26 Modernatx, Inc. Peg targeting compounds for delivery of therapeutics
WO2024206329A1 (en) 2023-03-27 2024-10-03 Modernatx, Inc. Nucleic acid molecules encoding bi-specific secreted engagers and uses thereof
WO2024206835A1 (en) 2023-03-30 2024-10-03 Modernatx, Inc. Circular mrna and production thereof
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
CN121463947A (zh) 2023-04-19 2026-02-03 赛欧生物医药股份有限公司 由包含rna和可电离脂质的脂质纳米颗粒递送多核苷酸
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
WO2024238723A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating pcsk9 expression
WO2024238726A1 (en) 2023-05-16 2024-11-21 Omega Therapeutics, Inc. Methods and compositions for modulating methylation of a target gene
WO2025019742A1 (en) 2023-07-19 2025-01-23 Omega Therapeutics, Inc. Methods and compositions for modulating ctnnb1 expression
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025042806A2 (en) 2023-08-21 2025-02-27 Modernatx, Inc. C-met binding antibodies, nucleic acids encoding same, and methods of use
WO2025054383A1 (en) 2023-09-06 2025-03-13 Modernatx, Inc. Chemical stability of mrna
WO2025059215A1 (en) 2023-09-12 2025-03-20 Aadigen, Llc Methods and compositions for treating or preventing cancer
WO2025059290A1 (en) 2023-09-14 2025-03-20 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
WO2025072482A1 (en) 2023-09-27 2025-04-03 Modernatx, Inc. Immunoglobulin a protease polypeptides, polynucleotides, and uses thereof
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025111297A1 (en) 2023-11-21 2025-05-30 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2025171182A1 (en) 2024-02-08 2025-08-14 Iovance Biotherapeutics, Inc. Treatment of cancer patients with tumor infiltrating lymphocyte therapies in combination with cancer vaccine
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025184429A1 (en) 2024-02-28 2025-09-04 Modernatx, Inc. Bromodomain and extra-terminal domain (bet) epigenetic reader decoy
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025235563A1 (en) 2024-05-07 2025-11-13 Omega Therapeutics, Inc. Epigenetic modulation for upregulation of genes
WO2025255199A1 (en) 2024-06-05 2025-12-11 Modernatx, Inc. Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof
WO2026003754A1 (en) 2024-06-25 2026-01-02 Life Edit Therapeutics, Inc. Novel reverse transcriptases and uses thereof
WO2026025031A1 (en) 2024-07-26 2026-01-29 Modernatx, Inc. Mrna encoding mageb2 tcer molecule

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5633076A (en) * 1989-12-01 1997-05-27 Pharming Bv Method of producing a transgenic bovine or transgenic bovine embryo
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
DE10335833A1 (de) * 2003-08-05 2005-03-03 Curevac Gmbh Transfektion von Blutzellen mit mRNA zur Immunstimulation und Gentherapie
US10837020B2 (en) * 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
DK2459231T3 (en) * 2009-07-31 2016-09-05 Ethris Gmbh RNA with a combination of unmodified and modified nucleotides for protein expression
WO2011130624A2 (en) * 2010-04-16 2011-10-20 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
EP2600901B1 (en) * 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
US8710200B2 (en) * 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
EP3460064B8 (en) * 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)

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