Sernova’s Hemophilia A program has received Orphan Drug and Rare Pediatric Disease designations from the FDA. This program involves the integration of Sernova’s Cell Pouch with a patient’s cells, eliminating the need for immunosuppression medications. The primary goal of this treatment approach is to substitute Factor VIII (FVIII), an essential protein for blood clotting that is absent in individuals with Hemophilia A.
The process encompasses the rectification of the patient’s own Blood Outgrowth Endothelial Cells (BOECs) and their subsequent reintroduction using the Cell Pouch. These modified cells are then responsible for releasing FVIII into the bloodstream, hence reinstating the patient’s ability to clot during bleeding episodes.
Sernova’s Collaboration Revolutionizing Hemophilia A Treatment
Sernova and its research collaborators have successfully corrected blood cells in individuals with Hemophilia A. This accomplishment was a result of an innovative gene and cell therapy, where these rectified cells were transplanted into a pre-implanted, vascularised Cell Pouch in a preclinical Hemophilia A model.
Sernova is cooperating with the University of Piemonte Orientale in Italy, under the leadership of Antonia Follenzi, Professor of Histology and Cell and Gene Therapy. The collaboration aims to enhance technology by utilizing lentiviral vectors to drive the expression of FVIII transgene. This is directed under the control of new promoters into BOECs of Hemophilia patients to achieve optimal sustained FVIII production, using an optimized cell dose within the Cell Pouch in an animal model of Hemophilia A.
Sernova’s CEO, Cynthia Pussinen, expressed the company’s commitment to advancing the program, emphasizing the serious and life-limiting nature of Hemophilia A. The aim is to have a positive impact on patients worldwide who are awaiting improved treatments.
Horizon 2020-Funded Hemophilia A Program
Sernova’s work is supported by a Horizon 2020 grant under the EU’s research and innovation funding program. The company’s Hemophilia A program is a significant step forward in the field of gene and cell therapy.
The Hemophilia A program by Sernova signifies a major advancement in the domain of gene and cell therapy. By integrating the patient’s cells with Sernova’s Cell Pouch, the reliance on immunosuppression medications is eliminated. The program’s main objective is to replace the blood clotting protein, Factor VIII, that is missing in Hemophilia A patients. The collaboration with the University of Piemonte Orientale in Italy aims to further optimize this technology, potentially bringing a significant positive impact to patients worldwide.
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