Neurogene Inc., a biotech company committed to developing life-changing genetic medicines for patients and their
families affected by rare neurological disorders, is excited to share an important update on their development program
for CLN5, a subtype of Neuronal Ceroid Lipofuscinosis (NCL), or Batten disease.
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.
This first-in-human gene therapy clinical trial is the first of its kind. Enrolled participants will receive NGN-101 to the brain [through intracerebroventricular (ICV) administration] and the eye [through intravitreal (IVT) administration] to address both the neurodegeneration and vision loss associated with CLN5 Batten disease.