JP2019519601A5 - - Google Patents

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Publication number
JP2019519601A5
JP2019519601A5 JP2019512957A JP2019512957A JP2019519601A5 JP 2019519601 A5 JP2019519601 A5 JP 2019519601A5 JP 2019512957 A JP2019512957 A JP 2019512957A JP 2019512957 A JP2019512957 A JP 2019512957A JP 2019519601 A5 JP2019519601 A5 JP 2019519601A5
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JP
Japan
Prior art keywords
hours
glycero
mrna
pharmaceutical composition
phosphocholine
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Pending
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JP2019512957A
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English (en)
Japanese (ja)
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JP2019519601A (ja
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Publication date
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Priority claimed from PCT/US2017/033418 external-priority patent/WO2017201346A1/en
Publication of JP2019519601A publication Critical patent/JP2019519601A/ja
Publication of JP2019519601A5 publication Critical patent/JP2019519601A5/ja
Priority to JP2023023253A priority Critical patent/JP2023062097A/ja
Pending legal-status Critical Current

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JP2019512957A 2016-05-18 2017-05-18 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド Pending JP2019519601A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023023253A JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338161P 2016-05-18 2016-05-18
US62/338,161 2016-05-18
EP17382259 2017-05-09
EP17382259.4 2017-05-09
PCT/US2017/033418 WO2017201346A1 (en) 2016-05-18 2017-05-18 Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023023253A Division JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Publications (2)

Publication Number Publication Date
JP2019519601A JP2019519601A (ja) 2019-07-11
JP2019519601A5 true JP2019519601A5 (enExample) 2020-06-25

Family

ID=67223874

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019512957A Pending JP2019519601A (ja) 2016-05-18 2017-05-18 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
JP2023023253A Pending JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Family Applications After (1)

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JP2023023253A Pending JP2023062097A (ja) 2016-05-18 2023-02-17 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド

Country Status (3)

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US (2) US20200085916A1 (enExample)
JP (2) JP2019519601A (enExample)
MA (1) MA45032A (enExample)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK4023249T3 (da) 2014-04-23 2025-01-13 Modernatx Inc Nukleinsyrevacciner
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
US12150980B2 (en) 2015-07-30 2024-11-26 Modernatx, Inc. Concatemeric peptide epitope RNAs
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
MA46316A (fr) 2015-10-22 2021-03-24 Modernatx Inc Vaccin contre le cytomégalovirus humain
CN117731769A (zh) 2015-10-22 2024-03-22 摩登纳特斯有限公司 用于水痘带状疱疹病毒(vzv)的核酸疫苗
MA52645B1 (fr) 2015-10-22 2022-06-30 Modernatx Inc Vaccins contre le virus respiratoire
MD3386484T2 (ro) 2015-12-10 2022-11-30 Modernatx Inc Compoziții și metode de livrare a unor agenți terapeutici
WO2017106799A1 (en) 2015-12-17 2017-06-22 Modernatx, Inc. POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE
SG11201810162PA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding citrin for the treatment of citrullinemia type 2
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
MA45053A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
WO2017201348A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
MA45050A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
CN109937253B (zh) 2016-09-14 2023-06-30 摩登纳特斯有限公司 高纯度rna组合物及其制备方法
WO2018075980A1 (en) 2016-10-21 2018-04-26 Modernatx, Inc. Human cytomegalovirus vaccine
US10925958B2 (en) 2016-11-11 2021-02-23 Modernatx, Inc. Influenza vaccine
MA50335A (fr) 2016-12-08 2020-08-19 Modernatx Inc Vaccins à acide nucléique contre des virus respiratoires
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. Respiratory syncytial virus vaccine
AU2018234692B2 (en) * 2017-03-15 2022-06-02 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US20200030432A1 (en) 2017-03-17 2020-01-30 Modernatx, Inc. Zoonotic disease rna vaccines
EP3607074A4 (en) 2017-04-05 2021-07-07 Modernatx, Inc. REDUCTION OR ELIMINATION OF IMMUNE RESPONSES TO NON-INTRAVENOUS THERAPEUTIC PROTEINS, FOR EXAMPLE SUBCUTANEOUSLY
US11390854B2 (en) * 2017-05-09 2022-07-19 Fundacion Para La Investigacion Medica Aplicada Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
US11912982B2 (en) 2017-08-18 2024-02-27 Modernatx, Inc. Methods for HPLC analysis
US11866696B2 (en) 2017-08-18 2024-01-09 Modernatx, Inc. Analytical HPLC methods
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
MA50253A (fr) 2017-09-14 2020-07-22 Modernatx Inc Vaccins à arn contre le virus zika
WO2019126144A1 (en) 2017-12-22 2019-06-27 North Carolina State University Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same
US11911453B2 (en) 2018-01-29 2024-02-27 Modernatx, Inc. RSV RNA vaccines
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
EP4509118A3 (en) 2018-09-19 2025-05-14 ModernaTX, Inc. High-purity peg lipids and uses thereof
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
JP2022521094A (ja) 2019-02-20 2022-04-05 モデルナティエックス インコーポレイテッド 共転写キャッピング用rnaポリメラーゼバリアント
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
EP3938507A4 (en) 2019-03-11 2023-02-22 ModernaTX, Inc. PROCEDURE FOR IN VITRO FED BATCH TRANSCRIPTION
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
TW202204622A (zh) 2020-04-09 2022-02-01 大陸商蘇州艾博生物科技有限公司 針對冠狀病毒之核酸疫苗
CN117003658B (zh) 2020-04-09 2025-10-14 苏州艾博生物科技有限公司 脂质纳米颗粒组合物
JP2023522249A (ja) 2020-04-22 2023-05-29 ビオンテック・ソシエタス・エウロパエア コロナウイルスワクチン
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
US20220218622A1 (en) 2020-10-14 2022-07-14 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
US12329811B2 (en) 2021-01-11 2025-06-17 Modernatx, Inc. Seasonal RNA influenza virus vaccines
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
CN114469996B (zh) * 2021-12-23 2023-10-20 中国医学科学院医学生物学研究所 一种包含miR-135b-5p的外泌体及在抗轮状病毒感染中的应用
TW202404568A (zh) * 2022-04-07 2024-02-01 美商Rna免疫公司 用於藥物遞送之化合物及組合物
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1049487B1 (en) 1998-01-27 2002-05-22 Hemebiotech A/S Treatment of acute intermittent porphyria (aip) and other porphyric diseases
JP2003505057A (ja) 1999-07-27 2003-02-12 ヘム−バイオテック エイ/エス rhPBGDの製造及び急性間欠性ポルフィリン症(AIP)及び他のポルフィリン症の患者を処置するための新規な治療方法
MX2011003229A (es) * 2008-09-29 2011-09-06 Proyecto Biomedicina Cima Sl Terapia genica con porfobilinogeno desaminasa.
EP3403647A1 (en) 2009-12-01 2018-11-21 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
JP2013095755A (ja) * 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
HRP20221536T1 (hr) 2014-06-25 2023-02-17 Acuitas Therapeutics Inc. Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
DK3350157T3 (da) 2015-09-17 2022-02-14 Modernatx Inc Forbindelser og sammensætninger til intracellulær afgivelse af terapeutiske midler
PT3368507T (pt) * 2015-10-28 2023-02-07 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
WO2017106799A1 (en) 2015-12-17 2017-06-22 Modernatx, Inc. POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE
SG11201810162PA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding citrin for the treatment of citrullinemia type 2
MA45050A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
WO2017201348A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
MA45053A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
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

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