AGTC Announces Completion of Enrollment in the Two Highest Dose Groups of Its Ongoing 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, announced that it has completed enrollment in the two highest dose groups of its Phase 1/2 clinical trial evaluating the safety and efficacy of subretinal injection of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. The patients in these additional groups received a higher or highest dose of AGTC’s XLRP candidate.

“We expect that the information that will be obtained from the two additional dose groups will help to reinforce the data generated to date and previously reported in September 2019 and January 2020,” said Sue Washer, president and CEO of AGTC. “We plan to report interim data from these two new dose groups and to report top-line 12-month data for the first four dose groups in the second half of 2020 and intend to initiate a pivotal trial by the end of the year. Combined with the ongoing progress in our two achromatopsia clinical studies, we expect to have multiple data read-outs in 2020 that will build on the momentum we created in January.”

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