Sernova has secured orphan drug designation (ODD) and rare paediatric disease designation (RPDD) from the US Food and Drug Administration (FDA) for its Haemophilia A programme.
The Hemophilia A programme integrates the Sernova Cell Pouch with a patient’s cells, eliminating the need for immunosuppression medications.
This treatment aims to substitute Factor VIII (FVIII), a crucial protein for blood clotting that is lacking in individuals with Haemophilia A.
It involves correcting the patient’s own Blood Outgrowth Endothelial Cells (BOECs) and then reintroducing them to the patient using the Cell Pouch.
The modified cells perform the function of releasing FVIII into the bloodstream, thereby restoring the patient’s ability to clot during episodes of bleeding.
Sernova CEO Cynthia Pussinen said: “Haemophilia A is a serious, life limiting condition and we are committed to advancing development of the programme, with a hope to positively impact patients around the world who are waiting for improved treatments.”
Sernova and its research collaborators, supported by a Horizon 2020 grant under the EU’s research and innovation funding programme, have corrected blood cells in individuals with Hemophilia A.
This was achieved by employing an innovative gene and cell therapy, where the corrected cells were transplanted into the pre-implanted, vascularised Cell Pouch in a preclinical Hemophilia A model.
The company has partnered with the University of Piemonte Orientale in Italy, led by Antonia Follenzi, who serves as Professor of Histology and Cell and Gene Therapy.
The new collaboration aims to optimise technology by using lentiviral vectors for driving the expression of FVIII transgene under the control of new promoters into BOECs of Haemophilia patients to attain optimal sustained FVIII production using an optimised cell dose within the Cell Pouch in an animal model of Haemophilia A.