CA2647903C - Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith - Google Patents

Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith Download PDF

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Publication number
CA2647903C
CA2647903C CA2647903A CA2647903A CA2647903C CA 2647903 C CA2647903 C CA 2647903C CA 2647903 A CA2647903 A CA 2647903A CA 2647903 A CA2647903 A CA 2647903A CA 2647903 C CA2647903 C CA 2647903C
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Prior art keywords
group
protein
nonsense
amino acid
substituted
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Expired - Fee Related
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CA2647903A
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English (en)
French (fr)
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CA2647903A1 (en
Inventor
Neil G. Almstead
Guangming Chen
Samit Hirawat
Seongwoo Hwang
Gary M. Karp
Langdon Miller
Young-Choon Moon
Hongyu Ren
Jemes J. TAKASUGI
Ellen M. Welch
Richard G. Wilde
Paul Kennedy
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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Expired - Fee Related legal-status Critical Current
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    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245—Oxadiazoles
    • 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
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/415—1,2-Diazoles
    • A61K31/4162—1,2-Diazoles condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A—HUMAN NECESSITIES
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/46—8-Azabicyclo [3.2.1] octane; Derivatives thereof, e.g. atropine, cocaine
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  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Otolaryngology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Child & Adolescent Psychology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Psychiatry (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2647903A 2006-03-30 2007-03-29 Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith Expired - Fee Related CA2647903C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US78733306P 2006-03-30 2006-03-30
US60/787,333 2006-03-30
US81308506P 2006-06-12 2006-06-12
US60/813,085 2006-06-12
PCT/US2007/008268 WO2007117438A2 (en) 2006-03-30 2007-03-29 Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith

Publications (2)

Publication Number Publication Date
CA2647903A1 CA2647903A1 (en) 2007-10-18
CA2647903C true CA2647903C (en) 2016-12-13

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CA2647903A Expired - Fee Related CA2647903C (en) 2006-03-30 2007-03-29 Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith

Country Status (15)

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US (2) US9289398B2 (enExample)
EP (2) EP2387995A1 (enExample)
JP (2) JP5367563B2 (enExample)
CN (1) CN101505739A (enExample)
AU (1) AU2007235524B2 (enExample)
BR (1) BRPI0710213A2 (enExample)
CA (1) CA2647903C (enExample)
IL (2) IL194454A (enExample)
IN (1) IN2014CN04406A (enExample)
MX (1) MX2008012515A (enExample)
NO (1) NO20084488L (enExample)
NZ (2) NZ596965A (enExample)
RU (1) RU2462246C2 (enExample)
SG (1) SG170819A1 (enExample)
WO (1) WO2007117438A2 (enExample)

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