QRX-1011

QRX-1011 is an investigational antisense oligonucleotide (ASO) developed by ProQR Therapeutics for the treatment of Stargardt's disease, a genetic condition leading to progressive vision loss. The therapeutic specifically targets the c.5461-10T>C mutation in the *ABCA4* gene, which causes aberrant splicing and the subsequent deletion of a critical portion of the ABCA4 protein. QRX-1011 works by binding to the pre-mRNA and modulating the splicing process to ensure the inclusion of the missing sequence, thereby restoring the production of functional, wild-type ABCA4 protein. This precision medicine approach is designed to address the underlying genetic cause of the disease with the potential to halt or reverse its progression.