RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

Inclusion Criteria: * Age ≥3 years and \<18 years (at the time of signing informed consent), any gender * Genetic test and clinical confirmed diagnosis of SHANK3-related PMS * Meets diagnostic criteria for moderate or more severe Autism Spectrum Disorder (ASD) * Intelligence Quotient (IQ) score \<70 or Developmental Quotient (DQ) (excluding gross motor) average score \<70 * Willing to provide biological samples required for the study (e.g., blood, urine) * Consent to hospitalization for intracerebroventricular injection surgery * The holders of parental authority who are able to understand and willing to comply with study requirements and procedures, voluntarily participating and signing the informed consent Exclusion Criteria: A pediatric participant who meets any of the following criteria will be excluded from this study: * Previous or current participation in other PMS drug clinical trials or other AAV gene therapy clinical studies * Has known allergic constitution, including allergy or hypersensitivity to prednisone acetate, other glucocorticosteroids, their excipients, or local anesthetics * Subjects with status epilepticus within 3 months prior to enrollment * Subjects requiring invasive or non-invasive ventilatory support * Serum anti-AAV neutralizing antibody titer \>1:200 * Significant laboratory abnormalities: alanine aminotransferase (ALT), aspartate aminotransferase (AST), γ-glutamyl transferase (GGT) with any value above the upper limit of normal; total bilir…