New Potential Gene Therapy for Retinal Degeneration Granted ODD

Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It’s called OCU400 and it’s being investigated as a treatment for NR2E3 mutation-associated retinal degenerative disease. This is a type of inherited retinal disease (IRD). It results in impaired vision or blindness.


This treatment is especially unique because researchers believe that it could be used to treat a wide variety of IRDs. Most gene therapy investigations for IRDs so far have been designed to replace a single gene. OCU400 could potentially treat various IRDs caused by different mutations in the NR2E3 gene.

OCU400 uses an AAV to deliver a functional copy of NR2E3 to cells in the retina. The expression of this gene in the retina helps to stabilize cells and reset retinal homeostasis. Additionally, it could potentially halt photoreceptor degeneration.

So far, researchers have examined this gene therapy in mouse models. In these studies, OCU400 successfully was able to reverse progression of the IRD when delivered straight to the retinal cells.

Scientists believe that this treatment could potentially be effective in retinitis pigmentosa, Leber Congenital Amaurosis, and a variety of other degenerative retinal diseases.

Looking Forward

By 2020, Ocugen hopes to have initiated a Phase 1/2a clinical trial examining the effect of OCU400 in IRDs.

IRDs caused by NR2E3 mutations were first discovered 30 years ago. Finally, these patients could potentially have a new treatment option.

Stay tuned to hear updates on this upcoming trial and further investigations of OCU400 in other degenerative retinal conditions.

You can read more about this potential new gene therapy for IRDs here.

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