Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs


The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.

“[With] traditional gene therapy, when you're giving a functional gene, you're not looking at the entire system. There's a lot coming out in epigenetics... [showing] there are other factors which impact degeneration. You can give a functioning gene, but maybe there is a cascading effect going on. So, if you don't have any photoreceptors left 5 years after you do gene therapy, which is supposed to be a one-time therapy for life, then it won't function, it won't work. So that's where we come in, our therapy resets the homeostasis, and provides a healthy environment for cells to survive. That's really important. So that's why we believe this is a way to go with a gene agnostic approach.”

OCU400, Ocugen's 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), along with some positive preliminary data from patients with Leber congenital amaurosis (LCA) enrolling in the latest cohort. The therapy has been generally well-tolerated, with no serious adverse events (SAEs) among the patients who received the low and medium doses of OCU400, although there were SAEs 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 mostly resolved in days to weeks.

CGTLive spoke with Shankar Musunuri, PhD, chairman, chief executive officer, and cofounder, Ocugen, to learn more about the current treatment landscape for patients with RP and the unmet needs that remain within it. He also shared the potential advantages of using a gene-agnostic, modifier gene therapy as opposed to a traditional gene-replacement therapy.

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. September 13, 2023.
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