Life science investment company Syncona Ltd reported on Monday that its portfolio company, Beacon Therapeutics, an ophthalmic gene therapy company, has declared promising three-month data from the Phase 2 SKYLINE trial of its gene therapy, AGTC-501, for X-Linked Retinitis Pigmentosa (XLRP).

The data reveal a robust efficacy profile with a 75% response rate in the higher dose cohort, showcasing significant improvement in retinal sensitivity. The safety profile of AGTC-501 remains favorable with no clinically significant safety events reported.

Beacon plans to present 12-month data in H1 CY2024 and commence the pivotal Phase 2/3 VISTA trial for AGTC-501 in XLRP during the same period.

XLRP stems from mutations in the RPGR gene. AGTC-501 addresses both rod and cone loss, expressing the full-length RPGR protein.

The positive SKYLINE trial results contribute to Syncona's mission to extend and enhance human life by creating and building companies focused on transformative treatments for high unmet medical needs.

Beacon Therapeutics, supported by Syncona, plans to revolutionize the treatment landscape for retinal diseases, emphasizing gene therapy's potential to save and restore vision. AGTC-501, in Phase 2 trials, aims to address the full spectrum of photoreceptor damage in XLRP patients.

The FLORetina ICOOR 2023 Congress served as the platform for Beacon's presentation on 2 December 2023, highlighting the encouraging progress in AGTC-501 development.