Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases

Inclusion Criteria: * Confirmation of two pathogenic variants in the IGHMBP2 gene from a CLIA-certified lab * Pre-ambulant (not yet walking and less than 18 months) or ambulant (as defined by the ability to walk 10 meters without assistance) or non-ambulant (inability to walk more than 10 meters unassisted) * Ability to cooperate with functional assessments as per PI's discretion Exclusion Criteria: * Prior participation in a gene or cell therapy program for any kind. * Immunizations of any kind in the month prior to the study. * Active infection based on clinical observations * Serological…