RP-A501

RP-A501 is an investigational gene therapy developed for the treatment of Danon disease, a rare genetic disorder caused by mutations in the LAMP2 gene. The therapy consists of a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human LAMP2B transgene (AAV9.LAMP2B). Administered as a single intravenous infusion, RP-A501 delivers the functional LAMP2B gene to cardiac cells (cardiomyocytes), aiming to restore lysosome-associated membrane protein 2 expression and address the underlying cause of Danon disease. Clinical studies have shown that RP-A501 can lead to sustained LAMP2 protein expression in heart tissue and improvements or stabilization in cardiac structure and function over periods up to several years[1][5][6].

Brand names

RP-A501RP-A-501RP-A 501

Other names

Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene