SAR422459

SAR422459 is a gene therapy developed for the treatment of Stargardt disease, a juvenile-onset macular degeneration caused by mutations in the ABCA4 gene. The drug consists of a lentiviral vector containing the human ABCA4 gene (EIAV-ABCA4), designed to deliver and express functional copies of this gene in retinal cells. By replacing or supplementing defective ABCA4 protein function via subretinal injection, SAR4224459 aims to address the underlying genetic cause of Stargardt’s disease and potentially slow or stabilize vision loss. Clinical trials have shown that subretinal administration was generally well tolerated; however, some treated eyes exhibited exacerbation of retinal pigment epithelium atrophy. Development has been suspended following sponsor review[2][6][8].

Brand names

StarGen

Other names

ABCA4 gene therapyABCA-4 gene therapyABCA 4 gene therapyATP-binding transporter gene therapyStargardt disease gene therapy - Oxford BioMedica/Sanofi