OCU400, a gene therapy candidate, has received a second orphan drug designation from the FDA for the treatment of CEP290 mutation associated retinal disease, Ocugen announced in a press release.
The CEP290 mutation is associated with Leber congenital amaurosis, Bardet-Biedl syndrome, Joubert syndrome, Senior-Loken syndrome and Meckel-Grüber syndrome.
“Orphan designation for this indication supports the goal of our modifier gene therapy platform to treat a variety of inherited retinal diseases with a single gene therapy,” Shankar Musunuri, PhD, MBA, Ocugen chairman, CEO and co-founder, said in the release. “There are currently no approved therapies for patients with the CEP290 mutation, which leads to severe visual impairment that tends to progress slowly over time. There is a significant unmet medical need for these patients and an opportunity to improve their quality of life with this novel approach.”