WO2014153643A8 - Compositions and methods for use thereof in the treatment of aniridia - Google Patents

Compositions and methods for use thereof in the treatment of aniridia Download PDF

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Publication number
WO2014153643A8
WO2014153643A8 PCT/CA2014/000286 CA2014000286W WO2014153643A8 WO 2014153643 A8 WO2014153643 A8 WO 2014153643A8 CA 2014000286 W CA2014000286 W CA 2014000286W WO 2014153643 A8 WO2014153643 A8 WO 2014153643A8
Authority
WO
WIPO (PCT)
Prior art keywords
treatment
methods
nonsense mutation
aniridia
compositions
Prior art date
Application number
PCT/CA2014/000286
Other languages
French (fr)
Other versions
WO2014153643A1 (en
Inventor
Cheryl GREGORY-EVANS
Kevin GREGORY-EVANS
Jin Ying ZHAO
Kishor Wasan
Original Assignee
The University Of British Columbia
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 The University Of British Columbia filed Critical The University Of British Columbia
Publication of WO2014153643A1 publication Critical patent/WO2014153643A1/en
Publication of WO2014153643A8 publication Critical patent/WO2014153643A8/en

Links

Classifications

    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/7036—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin having at least one amino group directly attached to the carbocyclic ring, e.g. streptomycin, gentamycin, amikacin, validamycin, fortimicins
    • 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
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38—Cellulose; Derivatives thereof
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Inorganic Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Provided herein are methods for treating a nonsense mutation eye condition, the method comprising the topical administration of one or more nonsense mutation read-through agent(s) formulated for topical delivery to the eye of a subject in need thereof and topical formulations of one or more nonsense mutation read-through agent(s) for use in the treatment of a nonsense mutation eye condition.
PCT/CA2014/000286 2013-03-26 2014-03-26 Compositions and methods for use thereof in the treatment of aniridia WO2014153643A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361805253P 2013-03-26 2013-03-26
US61/805,253 2013-03-26

Publications (2)

Publication Number Publication Date
WO2014153643A1 WO2014153643A1 (en) 2014-10-02
WO2014153643A8 true WO2014153643A8 (en) 2014-11-06

Family

ID=51622304

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CA2014/000286 WO2014153643A1 (en) 2013-03-26 2014-03-26 Compositions and methods for use thereof in the treatment of aniridia

Country Status (1)

Country Link
WO (1) WO2014153643A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI695717B (en) * 2014-03-06 2020-06-11 美商 Ptc 治療公司 Pharmaceutical compositions and salts of a 1,2,4-oxadiazole benzoic acid
TN2017000097A1 (en) 2014-10-24 2018-07-04 Takeda Pharmaceuticals Co Heterocyclic compound
US20240024292A1 (en) * 2020-12-14 2024-01-25 Institut National de la Santé et de la Recherche Médicale Ataluren eye drop formulation

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8716321B2 (en) * 2005-04-08 2014-05-06 Ptc Therapeutics, Inc. Methods for dosing an orally active 1,2,4-oxadiazole
CL2007002743A1 (en) * 2006-09-25 2008-07-11 Pct Therapeutics Inc Soc Organ CRYSTALLINE FORM OF ACID 3- [5- (2-FLUOROPHENYL) - [1,2,4] OXADIAZOL-3-IL] -BENZOIC; PHARMACEUTICAL COMPOSITION THAT INCLUDES SUCH CRYSTAL FORM; AND ITS USE TO MODULATE MARN'S PREMATURE TRANSFER OR DECLINE TERMINATION, SUCH AS CARCICOMA.

Also Published As

Publication number Publication date
WO2014153643A1 (en) 2014-10-02

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