JP2022500443A - 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 - Google Patents

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 Download PDF

Info

Publication number
JP2022500443A
JP2022500443A JP2021514132A JP2021514132A JP2022500443A JP 2022500443 A JP2022500443 A JP 2022500443A JP 2021514132 A JP2021514132 A JP 2021514132A JP 2021514132 A JP2021514132 A JP 2021514132A JP 2022500443 A JP2022500443 A JP 2022500443A
Authority
JP
Japan
Prior art keywords
abcb4
mir
sequence
utr
seq
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
JP2022500443A5 (https=
JPWO2020056370A5 (https=
Inventor
パオロ・マルティーニ
ヴラディミール・プレスニャク
ジンソン・カオ
Original Assignee
モデルナティーエックス， インコーポレイテッド
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by モデルナティーエックス， インコーポレイテッド filed Critical モデルナティーエックス， インコーポレイテッド
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

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 true JP2022500443A (ja) 2022-01-04
JP2022500443A5 JP2022500443A5 (https=) 2022-09-26
JPWO2020056370A5 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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140275229A1 (en) * 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
WO2017001570A2 (en) * 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
WO2017099823A1 (en) * 2015-12-10 2017-06-15 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
WO2018144775A1 (en) * 2017-02-01 2018-08-09 Modernatx, Inc. Immunomodulatory therapeutic mrna compositions encoding activating oncogene mutation peptides

Family Cites Families (8)

* 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
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
EP3368089B1 (en) * 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
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

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140275229A1 (en) * 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
WO2017001570A2 (en) * 2015-06-30 2017-01-05 Ethris Gmbh Atp-binding cassette family coding polyribonucleotides and formulations thereof
WO2017099823A1 (en) * 2015-12-10 2017-06-15 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
WO2018144775A1 (en) * 2017-02-01 2018-08-09 Modernatx, Inc. Immunomodulatory therapeutic mrna compositions encoding activating oncogene mutation peptides

Also Published As

Publication number Publication date
EP3863645A4 (en) 2022-11-16
WO2020056370A9 (en) 2020-05-07
AU2019338557A1 (en) 2021-03-25
US20220054653A1 (en) 2022-02-24
EP3863645A1 (en) 2021-08-18
WO2020056370A1 (en) 2020-03-19
CA3111836A1 (en) 2020-03-19

Similar Documents

Publication Publication Date Title
JP7459172B2 (ja) リラキシンをコードするポリヌクレオチド
JP2022500443A (ja) 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
JP7210287B2 (ja) Ｉｉ型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド
EP3458105B1 (en) Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP7423522B2 (ja) 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
JP2023062097A (ja) 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
EP3896164A1 (en) Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
JP7423521B2 (ja) フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
JP2022532078A (ja) 皮膚及び創傷のための組成物並びにその使用の方法
JP7424976B2 (ja) プロピオン酸血症の治療用のプロピオニルＣｏＡカルボキシラーゼアルファ及びベータサブユニットをコードするポリヌクレオチド
WO2017201317A1 (en) Polyribonucleotides containing reduced uracil content and uses thereof
EP3458106A1 (en) Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
JP2022500436A (ja) 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
JP2025028860A (ja) クリグラー－ナジャー症候群の治療のためのウリジン二リン酸グリコシルトランスフェラーゼ１ファミリー、ポリペプチドａ１をコードするポリヌクレオチド
EP3846776A1 (en) Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220913

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220913

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230710

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231010

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240110

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240507

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240807

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241202