A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene

Key Inclusion Criteria: * Maximum age for inclusion is 30 months. * Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan disease). * Participant has biochemical, genetic, and clinical diagnosis of Canavan disease: * Elevated urinary NAA and * Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history. * Active clinical signs of Canavan disease * Participant is…