A retrospective study conducted at Manchester Royal Eye Hospital reported long-term functional and anatomical outcomes of Voretigene neparvovec-rzyl (VN) therapy in patients with biallelic RPE65 mutation. The study, presented at Euretina 2024, followed 13 eyes from 7 patients over an average of 22 months.

Patients exhibited meaningful improvements in light sensitivity and visual fields, with significant enhancement in navigational ability, particularly in low-light conditions. Mean best-corrected visual acuity increased from 1.03 to 1.19 LogMAR at the last follow-up. Full-field stimulus testing improved significantly, with a mean reduction from 3.9 dB to -18.25 dB. Despite initial improvements, some patients experienced partial regression of visual acuity gains, especially in those with more advanced baseline degeneration.

Adverse events included vitritis, anterior uveitis, macular edema, and loss of the ellipsoid zone in some patients.

The presented abstract from Euretina 2024 and a full list of author disclosures can be found here.