Methods and pharmaceutical composition for the treatment and the prevention of cardiomyopathy due to energy failure

Abstract

The present invention relates to a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

1 . A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. 
     
     
         2 . The method according to  claim 1  wherein the cardiomyopathy due to energy failure is a cardiomyopathy associated with Friedreich ataxia and the gene that can restore energy failure is the frataxin (FXN) encoding nucleic acid. 
     
     
         3 . The method according to  claim 1 , wherein said FXN encoding nucleic acid encodes for the amino acid sequence SEQ ID NO:2. 
     
     
         4 . The method according to  claim 1  wherein the vector of claim comprises the nucleic acid sequence SEQ ID NO: 1. 
     
     
         5 . The method according to  claim 1  wherein the vector is selected from the group consisting of adenovirus, retrovirus, herpesvirus and Adeno-Associated Virus (AAV) vectors. 
     
     
         6 . The method according to  claim 5  wherein the vector is an AAV vector. 
     
     
         7 . The method according to  claim 6  wherein the AAV vector is an AAV1, AAV2, AAV3, AAV4, AA5, AAV6, AAV7, AAV8, AAV9, AAVrh10 vector or any AAV derived vector. 
     
     
         8 . The method according to  claim 7  wherein the AAV vector is an AAVrh10 vector. 
     
     
         9 . The method according to  claim 1  wherein the vector is administered intracoronary or directly into the myocardium of the subject. 
     
     
         10 . The method according to  claim 1  wherein the vector is administered by intravenous injection. 
     
     
         11 . A pharmaceutical composition for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. 
     
     
         12 . A pharmaceutical composition according to  claim 11  wherein the cardiomyopathy due to energy failure is a cardiomyopathy associated with Friedreich ataxia and the gene that can restore energy failure is the frataxin (FXN) encoding nucleic acid. 
     
     
         13 . A method for reversing symptoms of cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid. 
     
     
         14 . A vector which comprises a nucleic acid sequence of a gene that can restore energy failure for use in treatment or prevention of cardiomyopathy due to energy failure in a subject in need thereof. 
     
     
         15 . A vector according to  claim 14  wherein the vector comprises a frataxin (FXN) encoding nucleic acid. 
     
     
         16 . A vector according to  claim 14  wherein the cardiomyopathy is a cardiomyopathy associated with Friedreich ataxia. 
     
     
         17 . A vector according to  claim 14  wherein the vector is an AAVrh10 vector. 
     
     
         18 . A vector which comprises a frataxin (FXN) encoding nucleic acid for reversing symptoms of cardiomyopathy associated with Friedreich ataxia in a subject in need thereof. 
     
     
         19 . A vector which comprises a frataxin (FXN) encoding nucleic acid for use in the treatment of a cardiomyopathy associated with Friedreich ataxia in a symptomatic subject in need thereof. 
     
     
         20 . A vector according to  claim 19  wherein the vector is an AAVrh10 vector.