Biotechnology company Neurogene Inc has recently announced the planned initiation of a Phase I/II clinical trial for CLN5 disease utilising both intracerebroventricular and intravitreal delivery routes38 and a clinical trial is underway for the intravitreal delivery of the CLN2 enzyme replacement therapy Brineura (ClinicalTrials.gov identifier: NCT05152914). This evidence concerns the gene TPP1 and neuronal ceroid lipofuscinosis 5.