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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JP2019512957A
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English (en)
Japanese (ja)
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JP2019519601A (ja
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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
EP17382259.4 2017-05-09
EP17382259 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)

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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 (cg-RX-API-DMAC7.html) 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

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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)

Country Link
US (2) US20200085916A1 (cg-RX-API-DMAC7.html)
JP (2) JP2019519601A (cg-RX-API-DMAC7.html)
MA (1) MA45032A (cg-RX-API-DMAC7.html)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS66380B1 (sr) 2014-04-23 2025-02-28 Modernatx Inc Vakcine nukleinske kiseline
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
WO2017020026A1 (en) 2015-07-30 2017-02-02 Modernatx, Inc. Concatemeric peptide epitopes rnas
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
EP4349405A3 (en) 2015-10-22 2024-06-19 ModernaTX, Inc. Respiratory virus vaccines
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス, インコーポレイテッド ヒトサイトメガロウイルスワクチン
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
TN2018000152A1 (en) 2015-10-22 2019-10-04 Modernatx Inc Nucleic acid vaccines for varicella zoster virus (vzv)
WO2017099823A1 (en) 2015-12-10 2017-06-15 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
CA3007108A1 (en) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
MA45041A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1
SG11201810162PA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding citrin for the treatment of citrullinemia type 2
WO2017201333A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
US11801227B2 (en) 2016-05-18 2023-10-31 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
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
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
EP3528821A4 (en) 2016-10-21 2020-07-01 ModernaTX, Inc. HUMAN CYTOMEGALOVIRUS VACCINE
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
US11103578B2 (en) 2016-12-08 2021-08-31 Modernatx, Inc. Respiratory virus nucleic acid vaccines
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
RS63953B1 (sr) * 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
EP3607074A4 (en) 2017-04-05 2021-07-07 Modernatx, Inc. REDUCTION OR ELIMINATION OF IMMUNE RESPONSES TO NON-INTRAVENOUS THERAPEUTIC PROTEINS, FOR EXAMPLE SUBCUTANEOUSLY
WO2018206125A1 (en) * 2017-05-09 2018-11-15 Fundacion Para La Investigacion Medica Aplicada Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
EP3668977A4 (en) 2017-08-18 2021-04-21 Modernatx, Inc. HPLC ANALYTICAL PROCESSES
EP3668971B8 (en) 2017-08-18 2024-05-29 ModernaTX, Inc. Rna polymerase variants
EP3668979A4 (en) 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF HPLC ANALYSIS
CA3073211A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019055807A1 (en) 2017-09-14 2019-03-21 Modernatx, Inc. RNA VACCINES AGAINST ZIKA VIRUS
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
EP3852732A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Peg lipids and uses thereof
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
MA55037A (fr) 2019-02-20 2021-12-29 Modernatx Inc Variants d'arn polymérase pour le coiffage co-transcriptionnel
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
KR20210149727A (ko) 2019-03-11 2021-12-09 모더나티엑스, 인크. 유가식(fed-batch) 시험관내 전사 공정
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
WO2021202694A1 (en) * 2020-04-01 2021-10-07 Translate Bio, Inc. Phenolic acid lipid based cationic lipids
CN113874507A (zh) 2020-04-09 2021-12-31 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
CA3178455A1 (en) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Lipid nanoparticle composition
KR102813425B1 (ko) 2020-04-22 2025-05-28 바이오엔테크 에스이 코로나바이러스 백신
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
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
WO1999037325A2 (en) 1998-01-27 1999-07-29 Hemebiotech A/S Method for treating acute intermittent porphyria (aip) and other porphyric diseases
WO2001007065A2 (en) 1999-07-27 2001-02-01 Hemebiotech A/S PRODUCTION OF rhPBGD AND NEW THERAPEUTIC METHODS FOR TREATING PATIENTS WITH ACUTE INTERMITTENT PORPHYRIA (AIP) AND OTHER PORPHYRIC DISEASES
EA201170506A1 (ru) 2008-09-29 2011-12-30 Амстердам Молекьюлар Терапьютикс (Амт) Б.В. Генная терапия порфобилиногендезаминазой
US20110244026A1 (en) 2009-12-01 2011-10-06 Braydon Charles Guild 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
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
EP2834260A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
IL298516B2 (en) 2014-06-25 2025-03-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
ES2910425T3 (es) 2015-09-17 2022-05-12 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
SMT202300052T1 (it) * 2015-10-28 2023-05-12 Acuitas Therapeutics Inc Nuovi lipidi e for­mulazioni di nanoparticelle lipidiche per il rilascio di acidi nucleici
CA3007108A1 (en) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
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
US11801227B2 (en) 2016-05-18 2023-10-31 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
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
WO2017201333A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
MA45041A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1
SG11201810162PA (en) 2016-05-18 2018-12-28 Modernatx Inc Polynucleotides encoding citrin for the treatment of citrullinemia type 2

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