HUE031422T2 - Artificial 5'UTRs, expression vectors, and methods for increasing transgene expression - Google Patents
Artificial 5'UTRs, expression vectors, and methods for increasing transgene expression Download PDFInfo
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- HUE031422T2 HUE031422T2 HUE08833086A HUE08833086A HUE031422T2 HU E031422 T2 HUE031422 T2 HU E031422T2 HU E08833086 A HUE08833086 A HU E08833086A HU E08833086 A HUE08833086 A HU E08833086A HU E031422 T2 HUE031422 T2 HU E031422T2
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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4732—Casein
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0393—Animal model comprising a reporter system for screening tests
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/61—Fusion polypeptide containing an enzyme fusion for detection (lacZ, luciferase)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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Landscapes
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Families Citing this family (187)
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| US7723477B2 (en) | 2005-10-31 | 2010-05-25 | Oncomed Pharmaceuticals, Inc. | Compositions and methods for inhibiting Wnt-dependent solid tumor cell growth |
| PT2500360E (pt) | 2005-10-31 | 2015-10-15 | Oncomed Pharm Inc | Composições e métodos para diagnóstico e tratamento do cancro |
| US20090181458A1 (en) * | 2006-12-04 | 2009-07-16 | Thomas David Reed | Tubulo-vesicular structure localization signals |
| ES2614402T3 (es) | 2007-09-26 | 2017-05-31 | Intrexon Corporation | 5'UTR sintéticas, vectores de expresión y métodos para aumentar la expresión transgénica |
| ES2663536T3 (es) | 2008-09-26 | 2018-04-13 | Oncomed Pharmaceuticals, Inc. | Agentes que se unen a receptores frizzled y usos de los mismos |
| TWI535445B (zh) | 2010-01-12 | 2016-06-01 | 安可美德藥物股份有限公司 | Ｗｎｔ拮抗劑及治療和篩選方法 |
| CN102971337B (zh) | 2010-04-01 | 2016-09-21 | 昂考梅德药品有限公司 | 卷曲蛋白结合药剂及其应用 |
| US8822663B2 (en) | 2010-08-06 | 2014-09-02 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| DK4108671T3 (da) | 2010-10-01 | 2025-01-06 | Modernatx Inc | Modificerede nukleosider, nukleotider og nukleinsyrer samt anvendelser deraf |
| JP2014511687A (ja) | 2011-03-31 | 2014-05-19 | モデルナ セラピューティクス インコーポレイテッド | 工学操作された核酸の送達および製剤 |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| JP6113737B2 (ja) | 2011-10-03 | 2017-04-12 | モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． | 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法 |
| FR2981946B1 (fr) * | 2011-10-28 | 2015-02-20 | Lfb Biotechnologies | Unites de transcription et leur utilisation dans des vecteurs d'expression (yb2/0) |
| KR20140102759A (ko) | 2011-12-16 | 2014-08-22 | 모더나 세라퓨틱스, 인코포레이티드 | 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물 |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| HK1206612A1 (en) | 2012-04-02 | 2016-01-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of secreted proteins |
| EP2834259A4 (en) | 2012-04-02 | 2016-08-24 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| HK1212216A1 (en) | 2012-10-23 | 2016-06-10 | Oncomed Pharmaceuticals, Inc. | Methods of treating neuroendocrine tumors using wnt pathway-binding agents |
| US9597380B2 (en) | 2012-11-26 | 2017-03-21 | Modernatx, Inc. | Terminally modified RNA |
| CN105073995A (zh) * | 2013-02-01 | 2015-11-18 | 瑟莱克斯公司 | 增强的转基因表达和加工 |
| JP2016510411A (ja) | 2013-02-04 | 2016-04-07 | オンコメッド ファーマシューティカルズ インコーポレイテッド | Ｗｎｔ経路インヒビターによる処置の方法およびモニタリング |
| CN103131711B (zh) * | 2013-03-05 | 2014-05-14 | 中国农业科学院作物科学研究所 | 马铃薯pinⅡ基因5’UTR及其应用 |
| US9168300B2 (en) | 2013-03-14 | 2015-10-27 | Oncomed Pharmaceuticals, Inc. | MET-binding agents and uses thereof |
| WO2014144767A1 (en) | 2013-03-15 | 2014-09-18 | Moderna Therapeutics, Inc. | Ion exchange purification of mrna |
| US8980864B2 (en) | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| US11377470B2 (en) | 2013-03-15 | 2022-07-05 | Modernatx, Inc. | Ribonucleic acid purification |
| EP3578663A1 (en) | 2013-03-15 | 2019-12-11 | ModernaTX, Inc. | Manufacturing methods for production of rna transcripts |
| WO2014152030A1 (en) | 2013-03-15 | 2014-09-25 | Moderna Therapeutics, Inc. | Removal of dna fragments in mrna production process |
| DK3019619T3 (da) | 2013-07-11 | 2021-10-11 | Modernatx Inc | Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder |
| EP3041938A1 (en) | 2013-09-03 | 2016-07-13 | Moderna Therapeutics, Inc. | Circular polynucleotides |
| AU2014315287A1 (en) | 2013-09-03 | 2015-03-12 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| 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 |
| EP3052521A1 (en) | 2013-10-03 | 2016-08-10 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| GB201406006D0 (en) | 2014-04-03 | 2014-05-21 | Norwegian Univ Sci & Tech Ntnu | Synthetic mRNA leaders |
| US20180010136A1 (en) * | 2014-05-30 | 2018-01-11 | John Francis Hunt, III | Methods for Altering Polypeptide Expression |
| US10286086B2 (en) | 2014-06-19 | 2019-05-14 | Modernatx, Inc. | Alternative nucleic acid molecules and uses thereof |
| EP3169693B1 (en) | 2014-07-16 | 2022-03-09 | ModernaTX, Inc. | Chimeric polynucleotides |
| US10407683B2 (en) | 2014-07-16 | 2019-09-10 | Modernatx, Inc. | Circular polynucleotides |
| EP3171895A1 (en) | 2014-07-23 | 2017-05-31 | Modernatx, Inc. | Modified polynucleotides for the production of intrabodies |
| WO2017049286A1 (en) | 2015-09-17 | 2017-03-23 | Moderna Therapeutics, Inc. | Polynucleotides containing a morpholino linker |
| PL3350333T3 (pl) | 2015-09-17 | 2022-03-07 | Modernatx, Inc. | Polinukleotydy zawierające region stabilizujący ogon |
| AU2016336344A1 (en) | 2015-10-05 | 2018-04-19 | Modernatx, Inc. | Methods for therapeutic administration of messenger ribonucleic acid drugs |
| WO2017059902A1 (en) * | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| LT3394093T (lt) | 2015-12-23 | 2022-04-25 | Modernatx, Inc. | Ox40 ligandus koduojančių polinukleotidų naudojimo būdai |
| EP3400023A1 (en) | 2016-01-10 | 2018-11-14 | 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 |
| EP3442590A2 (en) | 2016-04-13 | 2019-02-20 | Modernatx, Inc. | Lipid compositions and their uses for intratumoral polynucleotide delivery |
| EP3458083B1 (en) | 2016-05-18 | 2022-11-02 | ModernaTX, Inc. | Polynucleotides encoding interleukin-12 (il12) and uses thereof |
| MA45052A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille |
| ES2973443T3 (es) | 2016-05-18 | 2024-06-20 | Modernatx Inc | Polinucleótidos que codifican galactosa-1-fosfato uridililtransferasa para el tratamiento de galactosemia de tipo 1 |
| CA3024624A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria |
| HRP20221135T8 (hr) | 2016-05-18 | 2023-02-03 | Modernatx, Inc. | KOMBINACIJE mRNK KOJE KODIRAJU IMUNOMODULIRAJUĆE POLIPEPTIDE I NJIHOVE UPOTREBE |
| EP3458081A1 (en) | 2016-05-18 | 2019-03-27 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| US20190298657A1 (en) | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| CA3024507A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding .alpha.-galactosidase a for the treatment of fabry disease |
| US12385034B2 (en) | 2016-06-24 | 2025-08-12 | Modernatx, Inc. | Methods and apparatus for filtration |
| IL266358B2 (en) | 2016-11-09 | 2025-10-01 | Precigen Inc | Constructs for Prataxin Expression |
| WO2018170473A1 (en) * | 2017-03-17 | 2018-09-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression |
| US11421011B2 (en) | 2017-05-18 | 2022-08-23 | Modernatx, Inc. | Polynucleotides encoding tethered interleukin-12 (IL12) polypeptides and uses thereof |
| US11485972B2 (en) | 2017-05-18 | 2022-11-01 | Modernatx, Inc. | Modified messenger RNA comprising functional RNA elements |
| WO2018231990A2 (en) | 2017-06-14 | 2018-12-20 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| EP3638292A1 (en) | 2017-06-14 | 2020-04-22 | ModernaTX, Inc. | Polynucleotides encoding coagulation factor viii |
| US20200254086A1 (en) | 2017-08-18 | 2020-08-13 | Moderna TX, Inc. | Efficacious mrna vaccines |
| 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 |
| WO2019104195A1 (en) | 2017-11-22 | 2019-05-31 | Modernatx, Inc. | Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia |
| US11802146B2 (en) | 2018-01-05 | 2023-10-31 | Modernatx, Inc. | Polynucleotides encoding anti-chikungunya virus antibodies |
| WO2019148101A1 (en) | 2018-01-29 | 2019-08-01 | Modernatx, Inc. | Rsv rna vaccines |
| EP3773745A1 (en) | 2018-04-11 | 2021-02-17 | ModernaTX, Inc. | Messenger rna comprising functional rna elements |
| US11875876B2 (en) | 2018-06-15 | 2024-01-16 | Massachusetts Institute Of Technology | Synthetic 5' UTR sequences, and high-throughput engineering and screening thereof |
| 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 |
| MA53545A (fr) | 2018-09-02 | 2021-07-14 | Modernatx Inc | Polynucléotides codant pour l'acyl-coa déshydrogénase à très longue chaîne pour le traitement de l'insuffisance en acyl-coa déshydrogénase à très longue chaîne |
| MX2021003015A (es) | 2018-09-13 | 2021-07-15 | Modernatx Inc | Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis. |
| EP3849594A2 (en) | 2018-09-13 | 2021-07-21 | Modernatx, Inc. | Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease |
| AU2019339430A1 (en) | 2018-09-14 | 2021-04-29 | Modernatx, Inc. | Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide A1 for the treatment of Crigler-Najjar Syndrome |
| WO2020056304A1 (en) | 2018-09-14 | 2020-03-19 | Modernatx, Inc. | Methods and compositions for treating cancer using mrna therapeutics |
| MA53734A (fr) | 2018-09-27 | 2021-08-04 | Modernatx Inc | Polynucléotides codant pour l'arginase 1 pour le traitement d'une déficience en arginase |
| JP2022531461A (ja) | 2019-05-07 | 2022-07-06 | モデルナティエックス インコーポレイテッド | 免疫細胞活性を破壊するポリヌクレオチド及びその使用方法 |
| WO2020227537A1 (en) | 2019-05-07 | 2020-11-12 | Modernatx, Inc | Differentially expressed immune cell micrornas for regulation of protein expression |
| JP2022532078A (ja) | 2019-05-08 | 2022-07-13 | アストラゼネカ アクチボラグ | 皮膚及び創傷のための組成物並びにその使用の方法 |
| US20220387628A1 (en) | 2019-06-24 | 2022-12-08 | Modernatx, Inc. | Messenger rna comprising functional rna elements and uses thereof |
| MA56539A (fr) | 2019-06-24 | 2022-04-27 | Modernatx Inc | Arn messager résistant à l'endonucléase et utilisations correspondantes |
| 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 |
| AU2021213108A1 (en) | 2020-01-28 | 2022-08-18 | Modernatx, Inc. | Coronavirus RNA vaccines |
| KR20220140528A (ko) | 2020-02-07 | 2022-10-18 | 모더나티엑스, 인크. | Sars-cov-2 mrna 도메인 백신 |
| TW202146432A (zh) * | 2020-04-03 | 2021-12-16 | 日商第一三共股份有限公司 | 新基因表現單元 |
| WO2021222304A1 (en) | 2020-04-27 | 2021-11-04 | Modernatx, Inc. | Sars-cov-2 rna vaccines |
| WO2021231854A1 (en) | 2020-05-14 | 2021-11-18 | Modernatx, Inc. | Lnp compositions comprising an mrna therapeutic and an effector molecule |
| WO2021159130A2 (en) | 2020-05-15 | 2021-08-12 | Modernatx, Inc. | Coronavirus rna vaccines and methods of use |
| EP4157217A1 (en) | 2020-06-01 | 2023-04-05 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof |
| US20230235298A1 (en) | 2020-06-01 | 2023-07-27 | Modernatx, Inc. | Phenylalanine hydroxylase variants 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 |
| US20230355743A1 (en) | 2020-09-25 | 2023-11-09 | Modernatx, Inc. | Multi-proline-substituted coronavirus spike protein vaccines |
| US20230406895A1 (en) | 2020-11-13 | 2023-12-21 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| US12329811B2 (en) | 2021-01-11 | 2025-06-17 | Modernatx, Inc. | Seasonal RNA influenza virus vaccines |
| CA3208303A1 (en) | 2021-01-15 | 2022-07-21 | Modernatx, Inc. | Variant strain-based coronavirus vaccines |
| AU2022208057A1 (en) | 2021-01-15 | 2023-08-03 | Modernatx, Inc. | Variant strain-based coronavirus vaccines |
| JP2024506908A (ja) | 2021-02-12 | 2024-02-15 | モデルナティエックス インコーポレイテッド | インビボ療法のためのペイロードを含むｌｎｐ組成物 |
| WO2022197624A1 (en) | 2021-03-15 | 2022-09-22 | Modernatx, Inc. | Therapeutic use of sars-cov-2 mrna domain vaccines |
| US20240216288A1 (en) | 2021-03-24 | 2024-07-04 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof |
| WO2022204390A1 (en) | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof |
| US20240226025A1 (en) | 2021-03-24 | 2024-07-11 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| WO2022204371A1 (en) | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof |
| US20240189449A1 (en) | 2021-03-24 | 2024-06-13 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency |
| AU2022249357A1 (en) | 2021-04-01 | 2023-10-12 | Modernatx, Inc. | Methods for identification and ratio determination of rna species in multivalent rna compositions |
| WO2022221440A1 (en) | 2021-04-14 | 2022-10-20 | Modernatx, Inc. | Influenza-coronavirus combination vaccines |
| WO2022245888A1 (en) | 2021-05-19 | 2022-11-24 | Modernatx, Inc. | Seasonal flu rna vaccines and methods of use |
| US20240269248A1 (en) | 2021-05-19 | 2024-08-15 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| WO2022266083A2 (en) | 2021-06-15 | 2022-12-22 | 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 |
| WO2023287751A1 (en) | 2021-07-12 | 2023-01-19 | Modernatx, Inc. | Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia |
| 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) |
| US20240368580A1 (en) | 2021-08-13 | 2024-11-07 | Modernatx, Inc. | Multicolumn chromatography mrna purification |
| AU2021461416A1 (en) | 2021-08-24 | 2024-02-22 | BioNTech SE | In vitro transcription technologies |
| US20250017867A1 (en) | 2021-10-01 | 2025-01-16 | Modernatx, Inc. | Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease |
| US20250281583A1 (en) | 2021-10-13 | 2025-09-11 | Modernatx, Inc. | Composition 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 |
| 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 |
| WO2023092069A1 (en) | 2021-11-18 | 2023-05-25 | Modernatx, Inc. | Sars-cov-2 mrna domain vaccines and methods of use |
| 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 |
| US20250360194A1 (en) | 2021-12-08 | 2025-11-27 | Modernatx, Inc. | Herpes simplex virus mrna vaccines |
| EP4452239A1 (en) | 2021-12-20 | 2024-10-30 | Sail Biomedicines, Inc. | Compositions comprising mrna and lipid reconstructed plant messenger packs |
| US20250084397A1 (en) | 2022-01-04 | 2025-03-13 | Modernatx, Inc. | Methods of purifying dna for gene synthesis |
| US20250115897A1 (en) | 2022-01-14 | 2025-04-10 | Modernatx, Inc. | In vitro transcription dna purification and recycling |
| CN118647718A (zh) | 2022-02-03 | 2024-09-13 | 摩登纳特斯有限公司 | 用于mRNA纯化的连续沉淀 |
| 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 |
| EP4499153A2 (en) | 2022-03-25 | 2025-02-05 | ModernaTX, Inc. | Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia |
| WO2023196399A1 (en) | 2022-04-06 | 2023-10-12 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria |
| WO2023196914A1 (en) | 2022-04-08 | 2023-10-12 | Modernatx, Inc. | Influenza nucleic acid compositions and uses thereof |
| EP4508069A1 (en) | 2022-04-15 | 2025-02-19 | ModernaTX, Inc. | Ribosomal engagement potency assay |
| WO2023215498A2 (en) | 2022-05-05 | 2023-11-09 | Modernatx, Inc. | Compositions and methods for cd28 antagonism |
| WO2023230481A1 (en) | 2022-05-24 | 2023-11-30 | Modernatx, Inc. | Orthopoxvirus vaccines |
| US20250388963A1 (en) | 2022-07-06 | 2025-12-25 | Modernatx, Inc. | Primer design for cell-free dna production |
| WO2024015890A1 (en) | 2022-07-13 | 2024-01-18 | Modernatx, Inc. | Norovirus mrna vaccines |
| WO2024050483A1 (en) | 2022-08-31 | 2024-03-07 | Modernatx, Inc. | Variant strain-based coronavirus vaccines and uses thereof |
| WO2024083345A1 (en) | 2022-10-21 | 2024-04-25 | BioNTech SE | Methods and uses associated with liquid compositions |
| EP4612301A1 (en) | 2022-11-03 | 2025-09-10 | ModernaTX, Inc. | Chemical stability of mrna |
| WO2024102434A1 (en) | 2022-11-10 | 2024-05-16 | Senda Biosciences, Inc. | Rna compositions comprising lipid nanoparticles or lipid reconstructed natural messenger packs |
| 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 |
| WO2024151811A1 (en) | 2023-01-11 | 2024-07-18 | Modernatx, Inc. | Personalized cancer vaccines |
| WO2024159172A1 (en) | 2023-01-27 | 2024-08-02 | Senda Biosciences, Inc. | A modified lipid composition and uses thereof |
| WO2024163465A1 (en) | 2023-01-30 | 2024-08-08 | Modernatx, Inc. | Epstein-barr virus mrna vaccines |
| 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 |
| AU2024235086A1 (en) | 2023-03-10 | 2025-09-11 | Modernatx, Inc. | Nucleic acid influenza vaccines and respiratory virus combination vaccines |
| EP4680287A1 (en) | 2023-03-15 | 2026-01-21 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| AU2024234874A1 (en) | 2023-03-15 | 2025-10-09 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2024197033A1 (en) | 2023-03-21 | 2024-09-26 | Modernatx, Inc. | Polynucleotides encoding relaxin for the treatment of heart failure |
| 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 |
| EP4694918A1 (en) | 2023-04-10 | 2026-02-18 | ModernaTX, Inc. | Lyme disease vaccines |
| WO2024220712A2 (en) | 2023-04-19 | 2024-10-24 | Sail Biomedicines, Inc. | Vaccine compositions |
| WO2024229321A1 (en) | 2023-05-03 | 2024-11-07 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| WO2024238726A1 (en) | 2023-05-16 | 2024-11-21 | Omega Therapeutics, Inc. | Methods and compositions for modulating methylation of a target gene |
| WO2024238723A1 (en) | 2023-05-16 | 2024-11-21 | Omega Therapeutics, Inc. | Methods and compositions for modulating pcsk9 expression |
| WO2024254552A1 (en) | 2023-06-08 | 2024-12-12 | Modernatx, Inc. | Stabilized flavivirus vaccines |
| WO2024263826A1 (en) | 2023-06-22 | 2024-12-26 | Modernatx, Inc. | Sars-cov-2 t cell vaccines |
| WO2025019352A2 (en) | 2023-07-14 | 2025-01-23 | Modernatx, Inc. | Mers-cov mrna vaccines |
| WO2025019742A1 (en) | 2023-07-19 | 2025-01-23 | Omega Therapeutics, Inc. | Methods and compositions for modulating ctnnb1 expression |
| WO2025029700A1 (en) | 2023-07-28 | 2025-02-06 | Modernatx, Inc. | Vlp enteroviral vaccines |
| WO2025034612A1 (en) | 2023-08-04 | 2025-02-13 | Modernatx, Inc. | Varicella-zoster virus mrna vaccine |
| WO2025042806A2 (en) | 2023-08-21 | 2025-02-27 | Modernatx, Inc. | C-met binding antibodies, nucleic acids encoding same, and methods of use |
| WO2025049959A2 (en) | 2023-09-01 | 2025-03-06 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025054383A1 (en) | 2023-09-06 | 2025-03-13 | Modernatx, Inc. | Chemical stability of mrna |
| WO2025059290A1 (en) | 2023-09-14 | 2025-03-20 | Modernatx, Inc. | Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria |
| AR133966A1 (es) * | 2023-09-27 | 2025-11-19 | Hanmi Pharmaceutical Co Ltd | UNA COMPOSICIÓN FARMACÉUTICA PARA PREVENIR O TRATAR EL CÁNCER QUE INCLUYE UN CONSTRUCTO DE ÁCIDO NUCLEICO QUE CODIFICA LA PROTEÍNA p53 |
| WO2025072482A1 (en) | 2023-09-27 | 2025-04-03 | Modernatx, Inc. | Immunoglobulin a protease polypeptides, polynucleotides, and uses thereof |
| WO2025081042A1 (en) | 2023-10-12 | 2025-04-17 | Renagade Therapeutics Management Inc. | Nickase-retron template-based precision editing system 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 |
| 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 |
| WO2025155753A2 (en) | 2024-01-17 | 2025-07-24 | Renagade Therapeutics Management Inc. | Improved gene editing system, guides, and methods |
| 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 |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025184429A1 (en) | 2024-02-28 | 2025-09-04 | Modernatx, Inc. | Bromodomain and extra-terminal domain (bet) epigenetic reader decoy |
| WO2025226656A1 (en) | 2024-04-23 | 2025-10-30 | Modernatx, Inc. | Hepatitis b virus mrna vaccines |
| CN119842704A (zh) * | 2024-05-06 | 2025-04-18 | 云舟生物科技(广州)股份有限公司 | 5'utr元件、表达载体及其应用 |
| WO2025235563A1 (en) | 2024-05-07 | 2025-11-13 | Omega Therapeutics, Inc. | Epigenetic modulation for upregulation of genes |
| WO2025250938A2 (en) | 2024-05-31 | 2025-12-04 | Modernatx, Inc. | Respiratory syncytial virus and metapneumovirus vaccines |
| WO2025255199A1 (en) | 2024-06-05 | 2025-12-11 | Modernatx, Inc. | Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof |
| WO2026025031A1 (en) | 2024-07-26 | 2026-01-29 | Modernatx, Inc. | Mrna encoding mageb2 tcer molecule |
| WO2026038592A1 (ko) * | 2024-08-14 | 2026-02-19 | (주)씨드모젠 | 신규한 합성 5'utr이 포함된 인공 핵산 분자 및 이의 용도 |
| WO2026044055A1 (en) | 2024-08-23 | 2026-02-26 | Modernatx, Inc. | Muc16 binding molecules, nucleic acids encoding same, and methods of use |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5354844A (en) | 1989-03-16 | 1994-10-11 | Boehringer Ingelheim International Gmbh | Protein-polycation conjugates |
| WO1993025567A1 (en) | 1992-06-15 | 1993-12-23 | Gene Pharming Europe B.V. | Production of recombinant polypeptides by bovine species and transgenic methods |
| US5633076A (en) * | 1989-12-01 | 1997-05-27 | Pharming Bv | Method of producing a transgenic bovine or transgenic bovine embryo |
| US7745416B2 (en) * | 1995-04-11 | 2010-06-29 | The Regents Of The University Of California | Method for in vivo regulation of cardiac muscle contractility |
| US7495087B2 (en) * | 1997-07-14 | 2009-02-24 | Bolder Biotechnology, Inc. | Cysteine muteins in the C-D loop of human interleukin-11 |
| US20030181405A1 (en) * | 1999-03-12 | 2003-09-25 | Nordstrom Jeffrey L. | Interferon alpha plasmids and delivery systems, and methods of making and using the same |
| BR0107930A (pt) * | 2000-01-28 | 2004-06-15 | Scripps Research Inst | Métodos de identificação de elementos reguladores transcricionais e translacionais sintéticos, e composições relativas aos mesmos |
| US6812339B1 (en) * | 2000-09-08 | 2004-11-02 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
| WO2002078642A2 (en) * | 2001-03-30 | 2002-10-10 | Origene Technologies, Inc | Differentially-expressed and up-regulated polynucleotides and polypeptides in breast cancer |
| US7179458B2 (en) * | 2002-03-08 | 2007-02-20 | Osel, Inc. | Lactobacilli expressing biologically active polypeptides and uses thereof |
| US7323306B2 (en) * | 2002-04-01 | 2008-01-29 | Brookhaven Science Associates, Llc | Genome signature tags |
| US7785871B2 (en) | 2002-10-09 | 2010-08-31 | Intrexon Corporation | DNA cloning vector plasmids and methods for their use |
| US20080050808A1 (en) | 2002-10-09 | 2008-02-28 | Reed Thomas D | DNA modular cloning vector plasmids and methods for their use |
| EP1751298A4 (en) | 2004-05-18 | 2009-11-11 | Intrexon Corp | METHOD FOR DYNAMIC VECTOR ASSEMBLY OF DNA CLONING VECTOR PLASMIDES |
| JP2008522589A (ja) * | 2004-12-03 | 2008-07-03 | ゾーマ テクノロジー リミテッド | 組換えタンパク質の発現のための方法及び材料 |
| GB0507997D0 (en) * | 2005-02-01 | 2005-05-25 | Powderject Vaccines Inc | Nucleic acid constructs |
| US9034650B2 (en) | 2005-02-02 | 2015-05-19 | Intrexon Corporation | Site-specific serine recombinases and methods of their use |
| WO2006089340A2 (en) * | 2005-02-23 | 2006-08-31 | V-Kardia Pty Ltd | Polynucleotide delivery to cardiac tissue |
| EP1871785B1 (en) * | 2005-04-20 | 2010-05-05 | Viromed Co., Ltd | Compositions and methods for fusion protein separation |
| US7491813B2 (en) * | 2005-12-07 | 2009-02-17 | Monsanto Technology Llc | Promoter polynucleotides identified from Zea mays for use in plants |
| KR20100049084A (ko) * | 2007-08-23 | 2010-05-11 | 인트렉손 코포레이션 | 질병 진단을 위한 방법 및 조성물 |
| ES2614402T3 (es) | 2007-09-26 | 2017-05-31 | Intrexon Corporation | 5'UTR sintéticas, vectores de expresión y métodos para aumentar la expresión transgénica |
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| US8835621B2 (en) | 2014-09-16 |
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