PT3463483T - Tratamento da discinesia ciliar primária com rna do mensageiro sintético - Google Patents

Tratamento da discinesia ciliar primária com rna do mensageiro sintético

Info

Publication number
PT3463483T
PT3463483T PT178036794T PT17803679T PT3463483T PT 3463483 T PT3463483 T PT 3463483T PT 178036794 T PT178036794 T PT 178036794T PT 17803679 T PT17803679 T PT 17803679T PT 3463483 T PT3463483 T PT 3463483T
Authority
PT
Portugal
Prior art keywords
treatment
messenger rna
ciliary dyskinesia
primary ciliary
synthetic messenger
Prior art date
Application number
PT178036794T
Other languages
English (en)
Portuguese (pt)
Original Assignee
Transcriptx Inc
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 Transcriptx Inc filed Critical Transcriptx Inc
Publication of PT3463483T publication Critical patent/PT3463483T/pt

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6925Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a microcapsule, nanocapsule, microbubble or nanobubble
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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 polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/06Antiasthmatics
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Pulmonology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nanotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
PT178036794T 2016-05-27 2017-05-26 Tratamento da discinesia ciliar primária com rna do mensageiro sintético PT3463483T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662342784P 2016-05-27 2016-05-27

Publications (1)

Publication Number Publication Date
PT3463483T true PT3463483T (pt) 2024-03-04

Family

ID=60411943

Family Applications (1)

Application Number Title Priority Date Filing Date
PT178036794T PT3463483T (pt) 2016-05-27 2017-05-26 Tratamento da discinesia ciliar primária com rna do mensageiro sintético

Country Status (10)

Country Link
US (6) US20190117796A1 (https=)
EP (2) EP3463483B1 (https=)
JP (3) JP7672786B2 (https=)
CN (2) CN117018228A (https=)
AU (3) AU2017271665B2 (https=)
CA (1) CA3025626A1 (https=)
DK (1) DK3463483T3 (https=)
ES (1) ES2972405T3 (https=)
PT (1) PT3463483T (https=)
WO (1) WO2017205767A1 (https=)

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CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
KR102832035B1 (ko) * 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
CA3100946A1 (en) 2018-05-22 2019-11-28 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
KR102116449B1 (ko) * 2018-10-01 2020-05-29 동국대학교 산학협력단 Ofd1을 이용한 피부 탈색 질환 치료용 조성물의 스크리닝 방법
JP2022516356A (ja) * 2019-01-07 2022-02-25 トランスレイト バイオ, インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
HUE065017T2 (hu) * 2019-02-14 2024-04-28 Ethris Gmbh Poliribonukleotid ciliogenezisre gyakorolt hatásának elemzése
JP7686573B6 (ja) 2019-05-24 2025-07-04 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド アルツハイマー病のための遺伝子治療
CN110438220A (zh) * 2019-08-16 2019-11-12 深圳市人民医院 纤毛不动综合症基因面板试剂盒及其应用
BR112022010373A2 (pt) * 2019-11-29 2022-08-16 Paros Bio Inc Terapia genética para doenças neurodegenerativas
CN111500697A (zh) * 2020-03-20 2020-08-07 四川省人民医院 不动纤毛综合症筛查试剂盒
EP4146680A1 (en) * 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス,インク. 細胞への標的送達のための組成物および方法
WO2022204215A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
JP2024527541A (ja) * 2021-07-01 2024-07-25 トランスレイト バイオ, インコーポレイテッド Mrnaの送達のための組成物
EP4379055A4 (en) * 2021-07-27 2025-08-06 Sk Bioscience Co Ltd MRNA FOR PROTEIN EXPRESSION AND TEMPLATE FOR IT
WO2023086893A1 (en) * 2021-11-10 2023-05-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
CN115287326B (zh) * 2022-08-05 2025-05-16 中国科学院深圳先进技术研究院 一种评估流感嗜血杆菌感染性的方法
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN119709879B (zh) * 2024-12-27 2025-09-26 天津大学 efcab2基因在构建脑积水动物模型中的应用及模型的构建方法

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JP2022516356A (ja) 2019-01-07 2022-02-25 トランスレイト バイオ, インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
HUE065017T2 (hu) 2019-02-14 2024-04-28 Ethris Gmbh Poliribonukleotid ciliogenezisre gyakorolt hatásának elemzése
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204215A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof

Also Published As

Publication number Publication date
AU2017271665A1 (en) 2019-01-17
EP4316588A2 (en) 2024-02-07
EP4316588A3 (en) 2024-05-15
WO2017205767A1 (en) 2017-11-30
JP2022120180A (ja) 2022-08-17
US20190117796A1 (en) 2019-04-25
AU2017271665B2 (en) 2023-11-23
US11642421B2 (en) 2023-05-09
JP7672786B2 (ja) 2025-05-08
DK3463483T3 (da) 2024-03-04
US20190111074A1 (en) 2019-04-18
JP2025090635A (ja) 2025-06-17
AU2025202461A1 (en) 2025-04-24
US20210162068A1 (en) 2021-06-03
AU2024200877A1 (en) 2024-02-29
CN109562192A (zh) 2019-04-02
US11786610B2 (en) 2023-10-17
CA3025626A1 (en) 2017-11-30
EP3463483A4 (en) 2019-12-18
US20220211873A1 (en) 2022-07-07
CN109562192B (zh) 2023-08-15
CN117018228A (zh) 2023-11-10
AU2024200877B2 (en) 2025-01-23
US20240285798A1 (en) 2024-08-29
JP2019520422A (ja) 2019-07-18
US20230190957A1 (en) 2023-06-22
ES2972405T3 (es) 2024-06-12
EP3463483A1 (en) 2019-04-10
US11510997B2 (en) 2022-11-29
EP3463483B1 (en) 2023-11-29

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