cell pouch with corrected patient cells (autologous)

cell pouch with corrected patient cells (autologous) is an investigational ex vivo gene-corrected cell therapy being developed by Sernova, in collaboration with the University of Piemonte Orientale, for the treatment of severe hemophilia A. The therapeutic approach involves isolating a patient's own blood outgrowth endothelial cells (BOECs), expanding them ex vivo, and using a lentiviral vector to insert a functional copy of the coagulation factor VIII (FVIII) gene. These gene-corrected autologous cells are subsequently transplanted into Sernova's proprietary, pre-vascularized, subcutaneously implanted Cell Pouch™ device. Once engrafted, the modified cells are designed to continuously produce and release functional FVIII into the bloodstream, restoring blood clotting capability and potentially offering a functional cure without the need for chronic systemic immunosuppression. The program has received Orphan Drug and Rare Pediatric Disease designations from the FDA and is currently in preclinical development.

Other names

Sernova hemophilia A cell therapySernova hemophilia A programSernova's Hemophilia A Cell Pouch System Programautologous gene-corrected BOECs in Cell Pouchex vivo lentiviral Factor VIII gene therapylentiviral vector-transduced BOECs in Cell Pouch