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

US10337027B2 · kind B2 · utility

Assignees

• APHP (ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS) · FR
• Cornell University · IT
• Institut National de la Sante et de la Recherche Medicale (INSERM) · FR
• CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE · FR
• UNIVERSITE DE STRASBOURG · 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: May 21, 2015
• Grant date: Jul 2, 2019
• Expiry date: May 21, 2035

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).