The FDA has granted a fourth orphan drug designation for OCU400 to treat PDE6B gene mutation-associated retinal diseases, according to an Ocugen press release.
The most recent designation targets retinitis pigmentosa caused by mutations in the PDE6B gene. RP stemming from this mutation leads to blindness by midlife and is characterized by the progressive loss of photoreceptors.
Ocugen’s modifier gene therapy platform allows ophthalmologists to address multiple diseases with a single therapy. Recently, OCU400 was granted an ODD to treat RHO mutation-associated retinal degeneration.
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