Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study.

Voretigene neparvovec (VN) is a gene therapy used to treat people with an inherited eye condition caused by changes in both copies of the RPE65 gene. The condition damages the retina and can lead to severe vision problems. The treatment is suitable only for people who still have enough healthy retinal cells.

The PERCEIVE study looked at how safe and effective this treatment was in real-world clinical practice. It included 103 patients, with an average age of around 20 years. Patients were followed for an average of about 10 months, although some were followed for more than two years.

Fischer MD, Simonelli F, Sahni J, Holz FG, Maier R, Fasser C, Suhner A, Stiehl DP, Chen B, Audo I, Leroy BP; PERCEIVE Study Group. Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study. Biomolecules. 2024 Jan 17;14(1):122. doi: 10.3390/biom14010122. PMID: 38254722; PMCID: PMC10813228.