A One-to-Many Approach to Gene Therapy for Retinal Disease

April 10, 2020

Gene therapies to treat retinal diseases promise to reverse blinding conditions, but the approach most gene developers take is to replace a mutated gene underlying a genetic disease with a functional version of that gene. The problem is that this approach can only address a narrow set of patients with each gene therapy. Ocugen, which Is developing gene therapies for eye diseases, believes it can treat a range of rare retinal conditions with a single therapy by introducing a functional gene that has the effect of modifying the expression of multiple genes at once. We spoke to Shankar Musunuri, CEO of Ocugen, about the company’s modifier gene therapy platform, how a single gene therapy can work on multiple eye diseases, and how it may alter the economics for gene therapy for rare eye conditions.

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