Ocugen gene therapy receives orphan drug designation

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

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