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Meeting on the Mesa 2021 | Treating achromatopsia with AAV-based gene therapy

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, describes the results of the preliminary Phase I/II trials of a subretinal adeno-associated virus (AAV)-based gene therapy in patients with achromatopsia, an ophthalmological condition affecting cone cells. In the trial assessing patients with a mutation in the CNGB3 gene (NCT02599922), a significant improvement in visual function was reported, whereas in the trial with patients with mutations in the CNGA3 gene (NCT02935517), the gene therapy did not increase visual acuity. This interview took place at Meeting on the Mesa 2021.