Canavan-Single Patient IND

• Male
• 18-24 months of age at time of study enrollment
• Have a diagnosis of Canavan disease, as defined by biochemical criteria AND/OR genetic mutation analysis, AND demonstrate clinical findings such as macrocephaly, developmental delay, seizures or other positive findings
• Elevated brain NAA levels, which is correlated with NAA acidemia and aciduria
• Willing to discontinue aspirin, aspirin-containing products and other drugs that may alter platelet function 7 days prior to dosing, resuming 24 hours after the gene transfer agent has been administered

Exclusion Criteria:

• Have required acute (as distinguished from long-term, maintenance or chronic suppressive) oral or intravenous antibiotic therapy for a respiratory infection within 15 days prior to screening
• Have required oral or systemic corticosteroids within the last 15 days prior to baseline screening
• Have a platelet count less than 75,000/mm3
• Have history of platelet dysfunction, evidence of abnormal platelet function at screening, or history of recent use of drugs that may alter platelet function, which the subject is unable/unwilling to discontinue for study agent administration
• Have an INR greater than 1.3
• Have transaminases and alkaline phosphatase more than ten times the upper limit of normal at screening or Day-1; or an abnormal chemistry profile
• Have bilirubin and gamma-glutamyl transpeptidase greater than 2 times the upper limit of normal at screening or Day -1
• Be currently or within the past 30 days participating in any other research protocol involving investigational agents or therapies (Other than approved therapy)
• Have received gene transfer agents within the past 6 months
• Have any other concurrent condition that, in the opinion of the investigator, would make the subject unsuitable for the study.