JP6506749B2 - フェニルケトン尿症のためのmRNA療法 - Google Patents

フェニルケトン尿症のためのmRNA療法 Download PDF

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JP6506749B2
JP6506749B2 JP2016523331A JP2016523331A JP6506749B2 JP 6506749 B2 JP6506749 B2 JP 6506749B2 JP 2016523331 A JP2016523331 A JP 2016523331A JP 2016523331 A JP2016523331 A JP 2016523331A JP 6506749 B2 JP6506749 B2 JP 6506749B2
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フランク デローザ,
フランク デローザ,
マイケル ハートレイン,
マイケル ハートレイン,
アヌシャ ディアス,
アヌシャ ディアス,
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シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド
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    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • 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
    • A61K48/0033Medicinal 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
    • 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
    • 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
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001Phenylalanine 4-monooxygenase (1.14.16.1)
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
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  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2016523331A 2013-10-22 2014-10-22 フェニルケトン尿症のためのmRNA療法 Active JP6506749B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361894303P 2013-10-22 2013-10-22
US61/894,303 2013-10-22
PCT/US2014/061830 WO2015061491A1 (en) 2013-10-22 2014-10-22 Mrna therapy for phenylketonuria

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JP2016535729A JP2016535729A (ja) 2016-11-17
JP2016535729A5 JP2016535729A5 (https=) 2017-11-30
JP6506749B2 true JP6506749B2 (ja) 2019-04-24

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JP2019066620A Pending JP2019135241A (ja) 2013-10-22 2019-03-29 フェニルケトン尿症のためのmRNA療法

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US (4) US9522176B2 (https=)
EP (2) EP3574923A1 (https=)
JP (2) JP6506749B2 (https=)
CN (1) CN105658242A (https=)
AU (1) AU2014340083B2 (https=)
CA (1) CA2928186A1 (https=)
EA (2) EA034103B1 (https=)
MX (1) MX2016005239A (https=)
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US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

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CA2928186A1 (en) 2015-04-30
US20230050301A1 (en) 2023-02-16
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JP2016535729A (ja) 2016-11-17
US9522176B2 (en) 2016-12-20
US12509666B2 (en) 2025-12-30
EP3060258A1 (en) 2016-08-31
EA034103B1 (ru) 2019-12-27
EA201690581A1 (ru) 2016-09-30
EP3574923A1 (en) 2019-12-04
CN105658242A (zh) 2016-06-08
US20190211314A1 (en) 2019-07-11
US20170073648A1 (en) 2017-03-16
JP2019135241A (ja) 2019-08-15
EA201992208A1 (ru) 2020-07-31
US11377642B2 (en) 2022-07-05
MX2016005239A (es) 2016-08-12
US10208295B2 (en) 2019-02-19
US20150110858A1 (en) 2015-04-23

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