Trial results presenting the safety and effectiveness of gene modifier therapy for treating retinitis pigmentosa associated with NR2E3 and RHO mutations.
- OCU400, a novel gene modifier therapy developed by Ocugen, has shown safety and effectiveness in treating retinitis pigmentosa associated with NR2E3 and RHO mutations.
- A phase 1/2 clinical trial of OCU400 in patients with retinitis pigmentosa demonstrated 89% of participants had stabilization or improvement in visual function measures.
- OCU400 delivers functional copies of the gene NR2E3 to retinal cells using an adeno-associated virus vector, resetting disrupted cellular gene networks and potentially improving retinal health and function.
- Safety assessments of OCU400 included monitoring adverse events, ophthalmologic changes, and immune and biochemical responses, with no serious adverse events reported in the low- and medium-dose groups.
- Efficacy assessments showed that 89% of participants experienced stabilization or improved visual function in the treated eye, with a phase 3 clinical trial currently underway for OCU400.
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Ophthalmology, Gene Therapy, Retinitis Pigmentosa.