Sernova’s Hemophilia A Program Receives Orphan Drug and Rare Pediatric Disease Designations from FDA
28 November 2023
Clinical-stage biotechnology company Sernova Corp. (Sernova) has announced that the FDA has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program. The company is also collaborating with the University of Peimonte Orientale.
Sernova is dedicated to developing therapeutic cell technologies for chronic diseases, with a primary focus on insulin-dependent diabetes and Hemophilia A. The company’s lead asset, the Cell Pouch System, serves as a novel implantable and scalable medical device containing immune-protected therapeutic cells.
Hemophilia A is a hereditary hemorrhagic disorder, that affects approximately 1 in 5,000 births, with 20,000 individuals in the US, 2,500 in Canada, and 10,000 in Europe experiencing moderate to severe forms. There is no cure, though regular infusions of recombinant clotting FVIII manage the condition. treatment costs per patient are high, reaching up to $200,000 or more annually, resulting in a total therapeutic cost exceeding $10 billion each year.
Sernova’s Hemophilia A program uses the Sernova Cell Pouch™ and a patient’s cells without needing immunosuppression medications. The Cell Pouch, when implanted, creates a natural environment for the long-term survival and function of therapeutic cells, which can release essential factors missing in certain chronic diseases. By correcting the patient’s Blood Outgrowth Endothelial Cells (BOECs) and returning them through the Cell Pouch™, modified cells release FVIII into the bloodstream, restoring the patient’s ability to clot during bleeding episodes.
“Hemophilia A is a serious, life-limiting condition and we are committed to advancing development of the program, with a hope to positively impact patients around the world who are waiting for improved treatments,” said Cynthia Pussinen, CEO of Sernova.
Sernova and research partners, through a Horizon 2020 grant which is part of the EU’s research and innovation funding program in proof-of-concept work, successfully corrected human blood cells from patients with hemophilia A to produce Factor VIII using a novel first-in-class gene and cell therapy approach where the corrected cells were transplanted into the pre-implanted, vascularized Cell Pouch in a preclinical model of Hemophilia A.
“We are pleased with the FDA’s decision to grant these designations for our novel treatment for Hemophilia A, which uses the Cell Pouch™ in combination with cells corrected for the production of Factor VIII,” added Pussinen.
Furthermore, Sernova is partnering with the University of Piemonte Orientale in Italy, led by Dr. Antonia Follenzi, to advance cell and gene therapy for Hemophilia A. The collaboration focuses on optimizing the use of lentiviral vectors to enhance the production of FVIII in BOECs from hemophilia patients. The goal is to achieve consistent and optimal FVIII expression in an animal model using the Cell Pouch. Successful pre-clinical results will pave the way for clinical trials to develop a treatment for Hemophilia A.
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