Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa
Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).
- Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).
- In addition, patients in the high dose cohort also demonstrated a robust improvement in visual function, including mean retinal sensitivity.
- XLRP is an orphan disease predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
- We look forward to announcing the initiation of our Phase 2/3 VISTA trial in the first half of 2024."