Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

US9066966B2 · kind B2 · utility

Assignees

• Institut National de la Sante et de la Recherche Medicale (INSERM) · FR
• CNRS-CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE · FR
• UNIVERSITE DE STRASBOURG · FR
• Cornell University · IT
• UNIVERSITÉ PARIS CITÉ · FR
• ASSISTANCE PUBLIQUE—HÔPITAUX DE PARIS (APHP) · FR

Inventors

• Hélène Puccio · Illkirch-Graffenstaden, FR
• Patrick Aubourg · Granville, US
• Ronald G. Crystal · Potomac, US
• Pierre Bougneres · Le Kremlin-Bicêtre, FR

Key dates

• Filing date: Feb 1, 2013
• Grant date: Jun 30, 2015
• Expiry date: Feb 1, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y116/03001
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).