JP2022500443A5 - - Google Patents

Info

Publication number
JP2022500443A5
JP2022500443A5 JP2021514132A JP2021514132A JP2022500443A5 JP 2022500443 A5 JP2022500443 A5 JP 2022500443A5 JP 2021514132 A JP2021514132 A JP 2021514132A JP 2021514132 A JP2021514132 A JP 2021514132A JP 2022500443 A5 JP2022500443 A5 JP 2022500443A5
Authority
JP
Japan
Prior art keywords
abcb4
lnp
orf
formulated
composition
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
Other languages
English (en)
Japanese (ja)
Other versions
JP2022500443A (ja
Filing date
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
Pending legal-status Critical Current

Links

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 (2)

Publication Number Publication Date
JP2022500443A JP2022500443A (ja) 2022-01-04
JP2022500443A5 true JP2022500443A5 (enExample) 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 (enExample)
EP (1) EP3863645A4 (enExample)
JP (1) JP2022500443A (enExample)
AU (1) AU2019338557A1 (enExample)
CA (1) CA3111836A1 (enExample)
WO (1) WO2020056370A1 (enExample)

Families Citing this family (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
MA42502A (fr) 2015-07-21 2018-05-30 Modernatx Inc Vaccins contre une maladie infectieuse
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
MA42543A (fr) 2015-07-30 2018-06-06 Modernatx Inc Arn épitope peptidiques concatémériques
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
EP4011451A1 (en) 2015-10-22 2022-06-15 ModernaTX, Inc. Metapneumovirus mrna vaccines
AU2016342045A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
WO2017070601A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (vzv)
AU2016341309A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Cancer vaccines
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
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
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
WO2018170256A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. RESPIRATORY SYNCYTIAL VIRUS Vaccine
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
EP3595676A4 (en) 2017-03-17 2021-05-05 Modernatx, Inc. RNA VACCINES AGAINST ZOONOSES
US11905525B2 (en) 2017-04-05 2024-02-20 Modernatx, Inc. Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
EP3668979A4 (en) 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF HPLC ANALYSIS
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019055807A1 (en) 2017-09-14 2019-03-21 Modernatx, Inc. RNA VACCINES AGAINST ZIKA VIRUS
EP3746090A4 (en) 2018-01-29 2021-11-17 ModernaTX, Inc. RSV RNA Vaccines
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061295A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. High-purity peg lipids and uses thereof
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
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
AU2020224103A1 (en) 2019-02-20 2021-09-16 Modernatx, Inc. Rna polymerase variants for co-transcriptional capping
CN113874502A (zh) 2019-03-11 2021-12-31 摩登纳特斯有限公司 补料分批体外转录方法
US12070495B2 (en) 2019-03-15 2024-08-27 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
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE 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
EP4274607A1 (en) 2021-01-11 2023-11-15 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
PL2578685T3 (pl) * 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
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
EP2906697A4 (en) * 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
US20160032316A1 (en) * 2013-03-14 2016-02-04 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
PL3386484T3 (pl) * 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
EP3426775A1 (en) * 2016-03-10 2019-01-16 Novartis AG Chemically modified messenger rna's
US12123030B2 (en) * 2016-05-18 2024-10-22 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
BR112019015797A2 (pt) * 2017-02-01 2020-03-17 Modernatx, Inc. Composições de mrna terapêuticas imunomoduladoras que codificam peptídeos de mutação de oncogene de ativação

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