Gene Therapy Study for Children With CLN5 Batten Disease

Exclusion Criteria

• •Has another neurologic disease or illness that may have caused cognitive decline before study entry
• •Known pathogenic or clinically suspected variant in a seizure associated genetic mutation besides CLN5
• •Any active infections or severe infections within the 30 days prior to study treatment administration
• •Presence of a concomitant medical condition that precludes intracerebroventricular (ICV) injection, lumbar puncture (LP), or use of anesthetics needed for study-related procedures
• •Presence of any concomitant medical conditions that preclude intravitreal (IVT) administration
• •Has status epilepticus that lasts longer than 5 minutes or having more than 1 seizure within a 5-minute period, without returning to a normal level of consciousness between episodes within 12 weeks before study treatment
• •Total anti-AAV9 antibody titer greater than 1:400
• •Any anticipated need for major surgery in the next 24 months
• •Participation in an Investigational New Drug, Investigational Device Exemption, or equivalent clinical study in the past 6 months
• •Any prior participation in a study in which a gene therapy vector or stem cell transplantation was administered
• •Participation in other investigational studies and non-interventional studies that have similar study assessments as this protocol while the study participant is enrolled in this study with the exception of sister studies sponsored by Neurogene
• •History of or current chemotherapy, radiotherapy, or other immunosuppressive therapy within the past 3 months
• •Use of prohibited medications
• •Immunizations of any kind in the 45 days prior to study treatment
• •Requiring daytime or nighttime ventilatory support at the time of Screening
• •Any item which would exclude the study participant from being able to undergo brain magnetic resonance imaging (MRI) according to local institutional policy
• •Known allergies or hypersensitivities to the required immunosuppression regime