We are very excited to share that the clinical trial for the investigational AAV (adeno-associated virus) gene therapy, NGN-101, is now enrolling participants at the clinical trial site, University of Rochester Medical Center in Rochester, NY, USA. This investigational gene therapy clinical trial is titled: A Phase 1/2 Intracerebroventricular and Intravitreal Administration of NGN-101 for Treatment of Neuronal Ceroid Lipofuscinosis (NCL) Subtype 5 (CLN5) Disease.
More details are in the Community Letter, and the link on clinicaltrials.gov is: NCT05228145
