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In episode 116 of Cell & Gene: The Podcast, Host Erin Harris talks to Dr. Arun Upadhyay, Chief Scientific officer, Head of Research and Development at Ocugen to discuss how the company’s modifier gene therapy platform is redefining treatment possibilities for inherited retinal diseases such as retinitis pigmentosa, Stargardt disease, and geographic atrophy. Dr. Upadhyay explains how Ocugen’s gene-agnostic approach differs from traditional single-gene therapies by targeting shared disease pathways to preserve photoreceptors and slow vision loss across diverse genetic mutations. He also shares key lessons learned from advancing OCU400, the first modifier gene therapy to receive a broad FDA indication for retinitis pigmentosa, including challenges in clinical trial design, endpoint selection, and manufacturing scalability.

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