Arun Upadhyay, PhD, on the Potential of Gene Therapy in Inherited Retinal Disease

“Our approach is more gene-agnostic in nature, which not only enhances the function of the photoreceptor, but also helps with preserving the surviving retinal cells through cellular and molecular homeostasis.”

Retinitis pigmentosa (RP) is an inherited retinal disease (IRD) that affects more than 100,000 people in the United States. Symptoms of RP include night blindness, a gradual loss of peripheral vision, and potentially blindness in the long-run. The condition is associated with over 100 genes. Although, currently the only targeted FDA-approved product for RP can only be used for the treatment of patients with a mutation in the RP65 gene; this represents only around 2% of patients with RP. As such, great unmet need remains for the overall patient population with RP. Because of the wide range of genes implicated in the condition, it is unlikely to be feasible for gene therapy approaches targeted at each specific gene to be developed and commercialized in a reasonable amount of time. In order to work around this issue, Ocugen has developed its Modifier Gene Therapy Platform, which may allow for a gene-agnostic approach to treating IRDs. One of the company’s gene therapy products based on this platform, OCU400, recently entered a phase 3 clinical trial (liMeliGhT; NCT06388200). The first patient in liMeliGhT was dosed in June of this year. Furthermore, findings from a phase 1/2 clinical trial (NCT05203939) in RP and Leber congenital amaurosis were recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.

In light of this milestone, CGTLive® reached out to Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the company’s approach. Upadhyay described the current landscape of care for RP and the unmet needs that remain. He then spoke about how OCU400 works and the design of the new phase 3 trial.

REFERENCES
1. Ocugen, Inc. Announces First Patient Dosed in Phase 3liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication. June 20, 2024. Accessed September 25, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-3-liMeliGhT-Clinical-Trial-for-OCU400-First-Gene-Therapy-in-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html