JPWO2020056370A5 - - Google Patents

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Publication number
JPWO2020056370A5
JPWO2020056370A5 JP2021514132A JP2021514132A JPWO2020056370A5 JP WO2020056370 A5 JPWO2020056370 A5 JP WO2020056370A5 JP 2021514132 A JP2021514132 A JP 2021514132A JP 2021514132 A JP2021514132 A JP 2021514132A JP WO2020056370 A5 JPWO2020056370 A5 JP WO2020056370A5
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JP
Japan
Prior art keywords
abcb4
lnp
orf
formulated
carrier
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2021514132A
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English (en)
Japanese (ja)
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JP2022500443A5 (https=
JP2022500443A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/051172 external-priority patent/WO2020056370A1/en
Publication of JP2022500443A publication Critical patent/JP2022500443A/ja
Publication of JP2022500443A5 publication Critical patent/JP2022500443A5/ja
Publication of JPWO2020056370A5 publication Critical patent/JPWO2020056370A5/ja
Pending legal-status Critical Current

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JP2021514132A 2018-09-13 2019-09-13 進行性家族性肝内胆汁うっ滞障害を処置するための修飾mRNA Pending JP2022500443A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862731055P 2018-09-13 2018-09-13
US62/731,055 2018-09-13
US201962840369P 2019-04-29 2019-04-29
US62/840,369 2019-04-29
PCT/US2019/051172 WO2020056370A1 (en) 2018-09-13 2019-09-13 Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders

Publications (3)

Publication Number Publication Date
JP2022500443A JP2022500443A (ja) 2022-01-04
JP2022500443A5 JP2022500443A5 (https=) 2022-09-26
JPWO2020056370A5 true JPWO2020056370A5 (https=) 2022-09-26

Family

ID=69778403

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021514132A Pending JP2022500443A (ja) 2018-09-13 2019-09-13 進行性家族性肝内胆汁うっ滞障害を処置するための修飾mRNA

Country Status (6)

Country Link
US (1) US20220054653A1 (https=)
EP (1) EP3863645A4 (https=)
JP (1) JP2022500443A (https=)
AU (1) AU2019338557A1 (https=)
CA (1) CA3111836A1 (https=)
WO (1) WO2020056370A1 (https=)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
EP3324979B1 (en) 2015-07-21 2022-10-12 ModernaTX, Inc. Infectious disease 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
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
TW201729835A (zh) 2015-10-22 2017-09-01 現代公司 呼吸道病毒疫苗
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
CA3002912A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (vzv)
HRP20220652T1 (hr) 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
MA45052A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille
CN116837052A (zh) 2016-09-14 2023-10-03 摩登纳特斯有限公司 高纯度rna组合物及其制备方法
MA46584A (fr) 2016-10-21 2019-08-28 Modernatx Inc Vaccin contre le cytomégalovirus humain
WO2018089851A2 (en) 2016-11-11 2018-05-17 Modernatx, Inc. Influenza vaccine
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
WO2018111967A1 (en) 2016-12-13 2018-06-21 Modernatx, Inc. Rna affinity purification
US11045540B2 (en) 2017-03-15 2021-06-29 Modernatx, Inc. Varicella zoster virus (VZV) vaccine
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. RESPIRATORY SYNCYTIAL VIRUS Vaccine
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
WO2018187590A1 (en) 2017-04-05 2018-10-11 Modernatx, Inc. Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
ES2983060T3 (es) 2017-08-18 2024-10-21 Modernatx Inc Variantes de ARN polimerasa
WO2019036685A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. METHODS FOR HPLC ANALYSIS
WO2019036683A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. ANALYTICAL METHODS BY HPLC
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
EP3681514A4 (en) 2017-09-14 2021-07-14 ModernaTX, Inc. RNA VACZINE AGAINST ZIKA VIRUS
WO2019148101A1 (en) 2018-01-29 2019-08-01 Modernatx, Inc. Rsv rna vaccines
MA53650A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg et leurs utilisations
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
CN113271926A (zh) 2018-09-20 2021-08-17 摩登纳特斯有限公司 脂质纳米颗粒的制备及其施用方法
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
CN113795579A (zh) 2019-02-20 2021-12-14 摩登纳特斯有限公司 用于共转录加帽的rna聚合酶变体
CA3132975A1 (en) 2019-03-11 2020-09-17 Modernatx, Inc. Fed-batch in vitro transcription process
EP3938379A4 (en) 2019-03-15 2023-02-22 ModernaTX, Inc. Hiv rna vaccines
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
EP3901261A1 (en) 2020-04-22 2021-10-27 BioNTech RNA Pharmaceuticals GmbH Coronavirus vaccine
WO2021231503A2 (en) * 2020-05-11 2021-11-18 The Board Of Trustees Of The Leland Stanford Junior University Systems and methods for enhancing gene expression
CN111744019B (zh) * 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
EP3936946A1 (fr) 2020-07-10 2022-01-12 Patek Philippe SA Genève Oscillateur horloger a pivot flexible
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
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
US12529047B1 (en) 2021-12-21 2026-01-20 Modernatx, Inc. mRNA quantification methods
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
CN115404273B (zh) * 2022-06-30 2023-06-23 湖南家辉生物技术有限公司 导致进行性家族性肝内胆汁淤积症ⅰ型的atp8b1基因突变体、蛋白和应用
WO2025024442A1 (en) * 2023-07-26 2025-01-30 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with bsep deficiency
WO2025212858A1 (en) 2024-04-03 2025-10-09 Children's Hospital Medical Center Ivt messenger rna and methods of treating or preventing pfic type iv
CN119587504B (zh) * 2024-12-10 2025-11-14 华中科技大学同济医学院附属协和医院 一种靶向骨组织巨噬细胞的纳米颗粒及其制备方法与应用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9012219B2 (en) * 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP3611266B1 (en) * 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
WO2011025862A2 (en) * 2009-08-28 2011-03-03 Curna, Inc. Treatment of atp-binding cassette sub-family b member 1 (abcb1) related diseases by inhibition of natural antisense transcript to abcb1
US20140275229A1 (en) * 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
WO2014062736A1 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
WO2014160243A1 (en) * 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
GB2560250A (en) * 2015-06-30 2018-09-05 Ethris Gmbh ATP-Binding cassette family coding polyribonucleotides and formulations thereof
EP3368089B1 (en) * 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
HRP20220652T1 (hr) * 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
US11377656B2 (en) * 2016-03-10 2022-07-05 Novartis Ag Chemically modified messenger RNA's
EP3458106A4 (en) * 2016-05-18 2020-03-18 Modernatx, Inc. POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA
JP2020514321A (ja) * 2017-02-01 2020-05-21 モデルナティーエックス, インコーポレイテッド 活性化がん遺伝子変異ペプチドをコードする免疫調節治療mRNA組成物

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