AGTC touts positive early clinical results for gene therapy

By The Science Advisory Board staff writers

Applied Genetic Technologies (AGTC) has reported positive data from its ongoing phase I/II clinical trial evaluating its adeno-associated virus (AAV)-based gene therapies in patients with X-linked retinitis pigmentosa (XLRP).

In the study, 62% of patients showed an improvement of at least 7 dB in at least 5 loci, which is the measure that the company is using to base statistically meaningful improvement between treated and untreated eyes. The company reported visual sensitivity, visual acuity, and safety data for six- and 12-month timepoints. Of the 15 evaluable patients in the treatment groups, 10 patients meet inclusion criteria for the planned Vista phase II/III trial, and show robust and durable signs of improvements in visual sensitivity through the sixth or twelfth month.

The company plans to initiate the phase II/III Vista clinical trial in early 2021 and to provide results from the six-month interim analysis in the third quarter of 2022. This trial will enroll patients from the phase I/II trial. AGTC will also begin a phase I/II expansion trial (Skyline) by the end of 2020.


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