Shankar Musunuri, PhD, on Promising Efficacy Signals With Modifier Gene-Agnostic Therapy in Retinitis Pigmentosa


The chairman, chief executive officer, and cofounder of Ocugen discussed the recent data update on the company’s OCU400 program in inherited retinal diseases.

“Rhodopsin mutations are very difficult to treat. That's why there's [no gene therapies] there. And this is one of the big ones causing RP, around 10,000 patients struggle with it in the US alone. So, in those patients, we showed very encouraging results 86% 6 out of 7 patients showed improvement. So that's important. Why? Because it's proving the concept of modifier genes... the gene agnostic approach.So if it works in rhodopsin, potentially it could work in all those patients I'm talking about, around 125 genetic mutations."

OCU400 (Ocugen) an investigational modifier gene therapy intended to treat inherited retinal diseases, has demonstrated improvement or stabilization of vision among patients with retinitis pigmentosa (RP) treated in a phase 1/2 clinical trial (NCT05203939). The new data announced also included some positive preliminary data from the latest cohort enrolling patients with Leber congenital amaurosis (LCA). The therapy has been generally well-tolerated, although there were serious adverse events (AEs) in 1 patient who received the high dose and 1 patient included in the open enrollment cohort with LCA. Most AEs were due to the surgical procedure and resolved in days to weeks.

CGTLive spoke with Shankar Musunuri, PhD, chairman, chief executive officer, and cofounder, Ocugen, to learn more about the updated data the company announced. He gave an overview of the positive findings on different outcome measures assessed in the study. He also stressed the advantages of modifier gene therapy compared to traditional gene therapy for rhodopsin mutations specifically.

Ocugen announces positive clinical study update from the phase 1/2 trial of OCU400, a modifier gene therapy product candidate, for the treatment of retinitis pigmentosa (R) and Leber congenital amaurosis (LCA). News release. Ocugen. September 13, 2023.
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