AAV-PHP.eB-aCD4LALAPG

AAV-PHP.eB-aCD4LALAPG is an investigational gene therapy designed for central nervous system (CNS)-targeted T-cell depletion in the treatment of multiple sclerosis (MS) [192]. It utilizes the CNS-tropic adeno-associated virus serotype PHP.eB (AAV-PHP.eB) vector to deliver a gene encoding an Fc-silenced anti-CD4 antibody construct (aCD4LALAPG) under the control of the neuron-specific human synapsin 1 (hSyn1) promoter [192]. By expressing the anti-CD4 antibody locally within neurons, the therapy aims to selectively deplete pathogenic CD4+ T cells in the CNS while minimizing peripheral T-cell depletion and associated systemic immunosuppression, thereby limiting neuroaxonal damage and disability progression [192].