SPK-601 is primarily utilized in the field of gene therapy, particularly for the treatment of inherited retinal diseases. It functions by delivering a functional copy of a specific gene to retinal cells, thereby addressing the underlying genetic defect. This approach aims to restore or preserve vision in patients suffering from conditions caused by mutations in the RPE65 gene. The therapy is administered through a subretinal injection, ensuring targeted delivery to the affected cells. Clinical trials have demonstrated its potential to improve visual function and slow disease progression, offering hope for individuals with limited treatment options.