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Meeting on the Mesa 2021 | AAV-based gene therapy for X-linked retinitis pigmentosa

Sue Washer, Applied Genetic Technologies Corporation, Alachua, FL, discusses the results of the Phase I/II and Phase II SKYLINE trials (NCT03316560) of a subretinal adeno-associated virus (AAV)-based gene therapy in patients with X-linked retinitis pigmentosa (XLRP). In the Phase I/II investigational portion of the trial, a majority of patients receiving a high dose reported an improvement in visual function and acuity, and in the SKYLINE trial, patients will receive further mobility tests to assess vision. An additional Phase III VISTA trial (NCT04850118) will be conducted to assess efficacy in another cohort. This interview took place at Meeting on the Mesa 2021.