Customized Gene Therapies Successfully Target Rare Eye Diseases

Can gene therapies be effective for the treatment of rare genetic diseases? One company may have found a solution that incorporates a high degree of customization, manufacturing expertise, and years’ worth of knowledge.

Having been with Applied Genetic Technologies Corporation (AGTC) for almost 20 years, CEO Sue Washer has first-hand experience in seeing novel ideas come into existence and leading the company through many developmental milestones. UF startup and UF Innovate | Sid Martin Biotech alum AGTC is a clinical-stage biotechnology company that focuses on gene therapies to restore visual function to patients with rare diseases. The company was founded in 1999 and has been working on these and other therapies ever since.

Washer explains that disease progression in the clinical indications the company is focusing on depends on the specifics of the genes that are mutated. The company’s lead product focuses on one such indication: X-linked retinitis pigmentosa (XLRP), a condition that is degenerative over time and usually results in complete blindness by the age of 60. Another disease that AGTC is targeting is achromatopsia. Individuals with the condition are born legally blind, with light sensitivity and no color vision, but their disease is stable over time.

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