We are so excited to share that the clinical trial for the investigational gene therapy for CLN5 disease is now enrolling participants at the trial site, University of Rochester Medical Center in Rochester, NY, USA.

This investigational clinical trial, sponsored by Neurogene is titled: A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease. Further information and trial details can be found at https://clinicaltrials.gov/ct2/show/NCT05228145...

We would like to thank and congratulate both Neurogene and the dedicated researchers who have together achieved this remarkable milestone. Their tireless work continues to bring hope to our Batten community.