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CLN5 News Feb 2022

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.

To learn more regarding the trials, you can find it here in the Community Letter. Or further details at the link on clinicaltrials.gov is: NCT05228145.

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