Beacon Therapeutics announced the presentation of interim safety and efficacy results from the phase 2 SKYLINE trial evaluating AGTC-501 gene therapy for X-linked retinitis pigmentosa.
At 12 months, 63% of study eyes treated with high-dose AGTC-501 experienced a response rate, defined as an improvement in retinal sensitivity of at least 7 dB in at least five loci, according to a company press release. Untreated eyes in the high-dose cohort and eyes that received low-dose AGTC-501 experienced a 0% response rate. Patients in the high-dose cohort also showed improvement in visual (Read more...)