Canavan-Single Patient IND

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.