IC-200

IC-200 is an investigational gene therapy being developed by Iveric Bio (now part of Astellas) for inherited retinal diseases caused by mutations in the BEST1 gene, including autosomal recessive bestrophinopathy and potentially other bestrophinopathies.[4][14] It is designed as a one-time subretinally delivered viral vector (likely an adeno-associated virus) carrying a functional copy of BEST1 to retinal pigment epithelial cells to restore or improve bestrophin-1 function, thereby correcting abnormal ion and fluid transport at the retinal pigment epithelium and aiming to preserve or improve visual function. Early development plans focus on patients with autosomal recessive bestrophinopathy, a severe form of BEST1-associated macular degeneration with limited treatment options.[4][14]