An adeno-associated virus-based gene therapy improved visual function in patients with X-linked retinitis pigmentosa 6 months after dosing, Applied Genetic Technologies Corporation announced in a press release.
Secondary data from a phase 1/2 clinical trial showed a favorable safety profile in all 25 patients treated with the AAV-based gene therapy product candidate. Patients treated centrally showed durable improvement in visual function.
“These promising results further demonstrate that our XLRP candidate has tremendous potential to provide meaningful benefit to XLRP patients who today have no treatment options,” Sue Washer, president and CEO of AGTC, said in the release. “The positive results observed to date give us confidence that the data as a whole will support advancement of our XLRP clinical program to a pivotal trial in 2020.”