Ocugen gene therapy receives orphan drug designation

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.

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.