AU2017268396B2 - Polynucleotides encoding citrin for the treatment of citrullinemia type 2 - Google Patents

Polynucleotides encoding citrin for the treatment of citrullinemia type 2 Download PDF

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Publication number
AU2017268396B2
AU2017268396B2 AU2017268396A AU2017268396A AU2017268396B2 AU 2017268396 B2 AU2017268396 B2 AU 2017268396B2 AU 2017268396 A AU2017268396 A AU 2017268396A AU 2017268396 A AU2017268396 A AU 2017268396A AU 2017268396 B2 AU2017268396 B2 AU 2017268396B2
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Australia
Prior art keywords
citrin
sequence
mrna
alkyl
codon
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Ceased
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AU2017268396A
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AU2017268396A1 (en
Inventor
Kerry BENENATO
Jinsong CAO
Lin Tung GUEY
Stephen Hoge
Ellalahewage Sathyajith Kumarasinghe
Paolo Martini
Iain Mcfadyen
Vladimir PRESNYAK
Staci SABNIS
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ModernaTx Inc
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ModernaTx Inc
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Priority to AU2023216799A priority Critical patent/AU2023216799A1/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—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
    • A61K38/00—Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Peptides Or Proteins (AREA)
AU2017268396A 2016-05-18 2017-05-18 Polynucleotides encoding citrin for the treatment of citrullinemia type 2 Ceased AU2017268396B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023216799A AU2023216799A1 (en) 2016-05-18 2023-08-16 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662338479P 2016-05-18 2016-05-18
US62/338,479 2016-05-18
PCT/US2017/033421 WO2017201349A1 (en) 2016-05-18 2017-05-18 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

Related Child Applications (1)

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AU2023216799A Division AU2023216799A1 (en) 2016-05-18 2023-08-16 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

Publications (2)

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AU2017268396A1 AU2017268396A1 (en) 2018-12-06
AU2017268396B2 true AU2017268396B2 (en) 2023-05-18

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AU2017268396A Ceased AU2017268396B2 (en) 2016-05-18 2017-05-18 Polynucleotides encoding citrin for the treatment of citrullinemia type 2
AU2023216799A Abandoned AU2023216799A1 (en) 2016-05-18 2023-08-16 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

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US (2) US10993918B2 (enExample)
EP (1) EP3458081A1 (enExample)
JP (2) JP7210287B2 (enExample)
AU (2) AU2017268396B2 (enExample)
CA (1) CA3024625A1 (enExample)
MA (1) MA45036A (enExample)
MY (1) MY201498A (enExample)
SG (1) SG11201810162PA (enExample)
WO (1) WO2017201349A1 (enExample)

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