MA49138A - Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr - Google Patents

Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr

Info

Publication number
MA49138A
MA49138A MA049138A MA49138A MA49138A MA 49138 A MA49138 A MA 49138A MA 049138 A MA049138 A MA 049138A MA 49138 A MA49138 A MA 49138A MA 49138 A MA49138 A MA 49138A
Authority
MA
Morocco
Prior art keywords
cftr
codon
administration
treatment
cystic fibrosis
Prior art date
Application number
MA049138A
Other languages
English (en)
Inventor
Jonathan Abysalh
Frank Derosa
Anusha Dias
Michael Heartlein
Shrirang Karve
Alan Kimura
Zarna Patel
Original Assignee
Translate Bio 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 Translate Bio Inc filed Critical Translate Bio Inc
Publication of MA49138A publication Critical patent/MA49138A/fr

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/12—Mucolytics
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Pulmonology (AREA)
  • Dispersion Chemistry (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Otolaryngology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
MA049138A 2017-05-16 2018-05-16 Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr MA49138A (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762507061P 2017-05-16 2017-05-16
US201762532301P 2017-07-13 2017-07-13
US201762580782P 2017-11-02 2017-11-02
US201762592238P 2017-11-29 2017-11-29
US201862659053P 2018-04-17 2018-04-17

Publications (1)

Publication Number Publication Date
MA49138A true MA49138A (fr) 2020-03-25

Family

ID=62567807

Family Applications (1)

Application Number Title Priority Date Filing Date
MA049138A MA49138A (fr) 2017-05-16 2018-05-16 Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr

Country Status (8)

Country Link
US (1) US11173190B2 (fr)
EP (1) EP3624824A1 (fr)
AU (1) AU2018268859A1 (fr)
CA (1) CA3063531A1 (fr)
IL (1) IL270631B2 (fr)
MA (1) MA49138A (fr)
MX (1) MX2019013752A (fr)
WO (1) WO2018213476A1 (fr)

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CA2838063C (fr) 2011-06-08 2023-07-11 Shire Human Genetic Therapies, Inc. Lipides clivables
MA45053A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
MX2019010155A (es) * 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
MX2021005969A (es) * 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
AU2020322014A1 (en) * 2019-07-30 2022-02-24 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
CA3162368A1 (fr) 2019-12-20 2021-06-24 Shrirang KARVE Administration par voie rectale d'arn messager
CN115461042A (zh) 2019-12-20 2022-12-09 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
WO2021142245A1 (fr) 2020-01-10 2021-07-15 Translate Bio, Inc. Composés, compositions pharmaceutiques et méthodes pour moduler l'expression de la muc5b dans des cellules et des tissus pulmonaires
EP4110296A1 (fr) 2020-02-25 2023-01-04 Translate Bio, Inc. Procédés améliorés de préparation de nanoparticules lipidiques chargées d'arnm
EP4138791A1 (fr) * 2020-04-20 2023-03-01 Board of Regents, The University of Texas System Compositions de poudre sèche biologiquement actives et leur procédé de fabrication et d'utilisation
EP4100539A4 (fr) 2020-04-27 2023-11-22 4D Molecular Therapeutics Inc. Variants adéno-associés, formulations et méthodes d'administration pulmonaire
EP4146342A1 (fr) * 2020-05-07 2023-03-15 Translate Bio, Inc. Compositions améliorées de thérapie par arnm de la cftr
AU2021273502A1 (en) 2020-05-15 2023-02-02 Translate Bio, Inc. Lipid nanoparticle formulations for mRNA delivery
CN116546976A (zh) 2020-10-06 2023-08-04 翻译生物公司 脂质纳米颗粒的改进工艺和配制
KR20230087536A (ko) 2020-10-12 2023-06-16 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 프로세스
WO2022081548A1 (fr) * 2020-10-12 2022-04-21 Translate Bio, Inc. Procédé amélioré de préparation de nanoparticules lipidiques à base de glace
WO2022099194A1 (fr) * 2020-11-09 2022-05-12 Translate Bio, Inc. Compositions améliorées pour l'administration d'arnm à optimisation des codons
US20220347112A1 (en) * 2021-03-31 2022-11-03 Rejuvenation Technologies Inc. Compositions and methods for delivery of rna
WO2022225918A1 (fr) 2021-04-19 2022-10-27 Translate Bio, Inc. Compositions améliorées pour l'administration d'arnm
CN117881395A (zh) 2021-07-01 2024-04-12 翻译生物公司 用于递送mRNA的组合物
AU2022381750A1 (en) * 2021-11-03 2024-05-16 Arcturus Therapeutics, Inc. Lipid formulations containing nucleic acids and methods of treatment for cystic fibrosis
CN114557971B (zh) * 2022-04-25 2023-05-23 康希诺生物股份公司 一种核酸-脂质纳米颗粒的冷冻干燥保护剂及其制备方法和应用

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