Sepofarsen: Investigational Treatment for Leber Congenital Amaurosis 10
Sepofarsen is an experimental mRNA therapy designed to improve visual function for patients with Leber congenital amaurosis 10. Sepofarsen targets a specific genetic mutation (c.2991+1655A>G) in the CEP290 gene. This mutation stops the cell from producing an essential protein needed for the cells in the retina to function. By addressing this mutation, sepofarsen aims to restore cell function in the retina. Sepofarsen is in Phase 3 clinical development.
Ultevursen: Investigational Treatment for Usher Syndrome Type 2a
Ultevursen is an experimental mRNA therapy designed to stabilize visual function for patients with Usher syndrome type 2a. Ultevursen targets specific gene mutations in the USH2 gene. These mutations stop the cell from producing usherin, an essential protein needed for the cells in the retina to function. By addressing this mutation, ultevursen aims to restore cell function in the retina. Ultevursen is in Phase 2 clinical development.
