INS1203

INS1203 is an investigational gene therapy being developed by Insmed for the treatment of Stargardt disease, a rare genetic eye disorder that causes progressive vision loss. Stargardt disease is most commonly caused by mutations in the *ABCA4* gene, which encodes a protein responsible for transporting retinoids in the retina; deficiency in this protein leads to the accumulation of toxic lipofuscin in the retinal pigment epithelium. INS1203 is designed to address the underlying genetic cause of the disease by delivering a functional version of the affected gene to retinal cells, potentially restoring normal lipid transport and preventing further photoreceptor degeneration. The program is currently in the preclinical stage of development.