The FDA has granted a third orphan drug designation for OCU400 to treat RHO mutation-associated retinal degeneration, according to a press release from Ocugen.
The mutation is part of the retinitis pigmentosa group of disorders. RHO mutations make up about 12% of patients with retinitis pigmentosa in the U.S.
OCU400 consists of a copy of a nuclear hormone receptor gene delivered to cells in the retina using an adeno-associated viral vector, the release said. When the gene is expressed in the retina, it may help reset retinal homeostasis, in addition (Read more...)