Sernova Announces Peer-Reviewed Publication Demonstrating the Safety and Efficacy of a Novel Pocket Cell Therapy Approach for the Treatment of Severe Hemophilia A
LONDON, ONTARIO – TheNewswire – January 20, 2022 – Sernova Corp. (TSXV:SVA) (OTC:SEOVF) (FSE/XETRA:PSH), a clinical-stage regenerative medicine and cell therapy company developing a potential “functional cure” for chronic disease is pleased to announce the release of a article in a peer-reviewed scientific journal highlighting the successful development of a new cell therapy approach for the treatment of severe hemophilia A, a serious genetic bleeding disorder caused by the absence of factor VIII (FVIII) activity in the bloodstream.
The journal article entitled “Efficient and Safe Correction of Hemophilia A by Lentiviral Vector-Transduced BOECs in an Implantable device (Sernova’s Cell Pouch™)”, was published in the prestigious scientific journal Molecular Therapy: Methods & Clinical Development, Volume 23, December 2021.
The therapeutic approach involves the use of the patient’s own cells obtained from a blood sample, which are then modified using a lentiviral vector-mediated gene transfer procedure using the form of FVIII domain-deleted cells under the control of an endothelial-specific promoter and then transplanted into Sernova’s vascularized cell pocket in a mouse model of hemophilia A. These cells then provide continuous therapeutic release of factor VIII into the bloodstream . The publication highlights that the HemAcure consortium has now succeeded in demonstrating safety and long-term improvement in blood clotting in a mouse model of hemophilia A.
An estimated 115,000 people live with hemophilia A in North America and Europe, a significant proportion of whom depend on regular infusions of FVIII. Treatment costs per patient amount to US$200,000 per year, with an overall therapeutic cost of over US$10 billion per year.
“This novel ex vivo gene therapy approach is the first demonstration showing the safety and feasibility of lentivirus-corrected blood outgrowth endothelial cell (BOEC) transplantation into an implantable medical device using GMP-like procedures for the long-term treatment of hemophilia A,” said lead author Dr. Antonia Follenzi, MD, PhD, professor, Department of Health Sciences, School of Medicine, Università del Piemonte Orientale. “If this therapy is successful in future clinical trials, it could become an important new therapeutic approach to improve the quality of life of people with severe hemophilia A.”
Dr. Philip Toleikis, President and CEO of Sernova Corp, said: “This publication represents approximately four years of dedicated work by the HemAcure Consortium, from conceptualizing this new treatment approach to validating its potential as a safe, long-term treatment option for people with hemophilia A. The Sernova cell pocket provides the required environment. for the transplanted cells to survive and function in the body, as evidenced by the production of FVIII to improve blood clotting, as reported by Dr. Follenzi and colleagues. He continued, “We recognize ex vivo gene therapy as a potential therapeutic option for people suffering from several rare diseases and we are proud that our technologies can contribute to the development and future delivery of functional cures for these diseases.
The HemAcure consortium research was supported by funding from the European Commission’s Horizon 2020.
ABOUT HEMOPHILIA A
Hemophilia A is the most common form of hemophilia and is a genetic disease caused by the reduction or absence of FVIII, a blood clotting protein. People with hemophilia A have prolonged abnormal bleeding following trauma or surgery. Although it is transmitted from parents to children, approximately 1/3 of cases are caused by a spontaneous modification of the gene responsible for the production of FVIII. According to the United States Centers for Disease Control and Prevention, hemophilia A occurs in approximately 1 in 5,000 male births. Prolonged bleeding in high-risk areas such as the brain can be catastrophic, while persistent bleeding in the joints lead to permanent damage, making joint replacement surgery common and risky in these patients. Approximately 115,000 people in North America and Europe have hemophilia A. Although there is no cure for the disease, hemophilia A can be controlled with medications to supplement or replace the missing FVIII . The annual worldwide costs for treating the disease for each patient can range from US$60,000 to US$260,000 for an overall therapeutic cost of approximately US$15 billion per year.
Sernova develops regenerative medicine therapeutic solutions using a medical device (Cell Pouch) and immunoprotected therapeutic cells/tissues (i.e. human donor cells, corrected human cells and stem cell-derived cells) to improve treatment and quality of life for people with metabolic diseases such as insulin-dependent diabetes, blood disorders including hemophilia, and other diseases treated by cellular production of proteins or hormones that are lacking or in insufficient quantity in the body. For more information, please visit www.sernova.com.
ABOUT SERNOVA CELL POUCH SYSTEM
The Cell Pouch, as part of the Cell Pouch System, is a novel, scalable, proprietary implantable macro-encapsulation device solution designed for the survival and long-term functioning of therapeutic cells. Upon implantation, the device is designed to embed itself into tissue, forming highly vascularized tissue chambers for transplantation and therapeutic cell function, which then release proteins and hormones as needed to treat disease.
The cell pocket, along with therapeutic cells, has been shown to provide long-term safety and efficacy in small and large animal models of diabetes and to provide a biologically compatible environment for cells that produce diabetes. insulin in humans in a Canadian first-in-man study. Sernova is currently conducting a phase I/II study at the University of Chicago. The first positive results were presented at several international scientific conferences.
ABOUT THE HORIZON 2020 PROGRAM AND HÉMACURE
Horizon 2020 was the biggest EU research and innovation program ever, with nearly €80 billion of funding available over seven years (2014-2020). It promised more breakthroughs, discoveries and world firsts by taking great ideas from lab to market. The project was funded as part of the “personalization of health and care” societal challenges in a specific call on innovative treatments and technologies. The overall objective of the HemAcure project was to develop and refine the tools and technologies of a new cell therapy prepared ex vivo to treat the bleeding disease, hemophilia A (caused by a genetic deficiency of coagulation factor VIII ( FVIII)) which should ultimately lead to a better quality of life for patients. The European Union’s Horizon 2020 research and innovation program has awarded around 5.5 million euros (C$8.5) to support the project. international consortium members included Würzburg University Hospital (Coordinating Institute), Germany; IMS – Integrierte Management, Heppenheim, Germany; Università del Piemonte Orientale “Amedeo Avogadro”, Novara, Italy; Loughborough University, Loughborough, UK; ARTTIC International Management Services, Munich, Germany and Sernova Corp., London, Ontario, Canada. This project has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement n° 667421. The consortium worked together to provide sets of design and manufacturing protocols, based on current European GMP regulations, to prepare an Investigational Medical Product Dossier (IMPD) for an Investigational Medical Product (IMP), consisting of cells therapies and an implantable medical device (Sernova’s Cell Pocket).
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Vice President, Investor Relations
Corey Davis, Ph.D.
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