AGTC Reports Additional Positive Data From Its Phase 1/2 Clinical Trial in Patients With X-Linked Retinitis Pigmentosa

UF startup and UF Innovate | Sid Martin Biotech alum Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, reported positive data from its ongoing Phase 1/2 clinical program in patients with X-linked retinitis pigmentosa (XLRP). Visual sensitivity, visual acuity and safety data were reported for 12-month timepoints for Groups 2 and 4, and 6-month time points for Groups 5 and 6. Eight of the 11 patients in Groups 5 and 6 would meet the eligibility criteria for AGTC’s future XLRP trials, and five of these eight (62%), met the definition for response based on an improvement of at least 7 decibels in at least 5 loci. The company plans to initiate its Skyline trial by enrolling approximately 12 additional patients total in Group 2 (1.2E+11 vg/mL) and Group 5 (1.1E+12 vg/mL). In addition, the company remains on track to initiate enrollment in its planned Vista trial in 1Q 2021.