SGX-003

SGX-003 is a discovery-stage ex vivo gene therapy being developed by Somagenetix, in collaboration with the University of Zurich (UZH), for the treatment of Frontotemporal Dementia (FTD). The program utilizes Somagenetix's proprietary LV-SIN (self-inactivating) lentiviral vector platform to genetically engineer a patient's own hematopoietic stem cells (HSCs). These modified stem cells are intended to engraft in the bone marrow and produce progeny, including microglia-like cells that migrate to the central nervous system to deliver a therapeutic transgene. While the specific molecular target of SGX-003 has not been publicly disclosed, the approach is designed to address neurodegenerative processes in the brain through localized expression of a corrective protein.