Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia

Inclusion Criteria

• •At the time of dosing, participants must be 6 months to 12 years of age
• •Males and females with diagnosis of severe MMA meeting all the following;
• •1. Isolated MMA with genetically confirmed, pathogenic mutations in the MMUT gene
• •2. Screening serum/plasma methylmalonic acid level of \>100 µmol/L
• •3. One or more of the following considered by the PI to be MMA-related: (i) An unscheduled ER visit, hospitalization or requirement for sick day diet in the year prior to screening visit (ii) Developmental delay, movement disorder, optic neuropathy or feeding disorder with tube feeding requirement
• •4. Medically stable for the 2 months prior to the start of screening