The FDA has granted orphan drug designation to OCU400, Ocugen’s gene therapy for the treatment of NR2E3 mutation-associated retinal degenerative disease, according to a press release.
OCU400 has been shown to deliver NR2E3 to retinal cells and reverse disease progression in a mouse model with NR2E3 mutation, the release said.
The company plans to begin a phase 1/2a study of the gene therapy by 2020.
“Unlike single-gene replacement approaches, which have shown great promise in rare retinal diseases despite being highly specific for a single condition, we believe OCU400 represents a powerful and broad means of treating a variety of genetically diverse [inherited retinal diseases] with a single product. Our initial strategy is to develop OCU400 as a gene augmentation therapy for patients with [inherited retinal diseases] caused by mutations in the NR2E3 gene,” Ocugen Chairman and CEO Shankar Musunuri, PhD, MBA, said in the release.