Mr. Dominic Gray reports
SERNOVA HIGHLIGHTS ACHIEVEMENTS OF THE HEMACURE HEMOPHILIA CELL THERAPY PROGRAM PRESENTED AT ASGCT
Sernova Corp. is highlighting positive results associated with the development of a novel, safe cell therapy approach, including its proprietary Cell Pouch, for the treatment of hemophilia A as presented on May 15, 2020, by the Horizon 2020 HemAcure consortium at the American Society of Gene and Cell Therapy's 23rd annual meeting (ASGCT).
The aim of the HemAcure three-year project was to develop a permanent safe therapeutic solution for those living with hemophilia A in the form of a novel ex vivo gene therapy, cell-based approach within Sernova's proprietary Cell Pouch. This combination therapy strives to permanently replace missing clotting human factor VIII (FVIII) in the patient's own Blood Outgrowth Endothelial Cells (BOECs) transplanted into the Cell Pouch. These corrected cells are to function to release FVIII into the bloodstream restoring the ability for blood clotting to occur during periods of bleeding.
The following are highlights of the results presented
- BOECs were safely isolated and grown from a small sample of circulating peripheral blood of volunteer Hemophilia A patients unable to express the required FVIII for clotting.
- To regain the function of the BOECs ability to produce clotting FVIII, techniques were successful in safely inserting the gene responsible for the correction and production of human FVIII into the patient's BOECs, and these corrected cells were safely multiplied to increase their number.
- Tests were conducted to ensure the safety, and the newly corrected BOECs produced enough human FVIII both in the laboratory and in an initial preclinical animal model deficient of FVIII. FVIII blood levels reached up to 10%, a therapeutically relevant level of FVIII.
- To further test cell dose-response, in the preclinical model of hemophilia A, animals originally unable to clot their blood were implanted with a small vascularized Cell Pouch and transplanted with two different doses of human BOECs corrected for the ability to produce human FVIII.
- To assess the safety of the combined product, the Cell Pouch and corrected human FVIII BOECs derived from the volunteer participants with hemophilia A were examined using histological analyses. Importantly, histology showed healthy tissue represented by the presence of stromal growth and new blood vessel formation within the Cell Pouch.
- Further, histological investigation of the transplanted Cell Pouch sections demonstrated long-term survival of human FVIII BOECs present within the vascularized Cell Pouch achieved through co-staining for blood vessels (von Willebrand Factor stain) and the presence of the patients corrected human cells (HLA-ABC stain).
- In both experimental doses, human FVIII was detected in circulating peripheral blood up to 16 weeks following transplantation, with more human FVIII present in peripheral blood using the higher dose of corrected BOECs.
- Data further confirmed functional clotting improvement in the blood at the four months time point where FVIII BOECs transplanted into the hemophilia A mouse model restored the animals FVIII activity at a therapeutic level in the Cell Pouch.
In summary, these results demonstrate the success of the HemAcure Consortium team in developing a novel approach for the treatment of hemophilia A using an ex vivo gene therapy, cell-based technology that could lead to improved efficacy and quality of life of people suffering from hemophilia A.
"These promising results of the HemAcure consortium are paving the way for future human clinical testing in hemophilia A patients using Sernova's technologies transplanted with corrected FVIII cells developed by this prestigious international team. This initiative continues to support the value of collaborative efforts involving Sernova's regenerative medicine technologies in both diabetes and now rare diseases such as hemophilia A," said Dr. Philip Toleikis, President and CEO of Sernova Corp.
The overall objective of the HemAcure Project is to develop and refine the tools and technologies for a novel ex vivo prepared cell-based therapy to treat the bleeding disease, hemophilia A (caused by a genetic deficiency in clotting factor VIII) that should ultimately lead to improved quality of life of the patients. The European Union's Horizon 2020 Research and Innovation program awarded approximately EUR5.5 million (~CDN$8.5 million) to support the project. International consortium members include the University Hospital Wurzburg (Coordinating Institute), Germany, IMS - Integrierte Management, Heppenheim, Germany; Universita del Piemonte Orientale "Amedeo Avogadro," Novara, Italy; Loughborough University, Loughborough, United Kingdom; ARTTIC International Management Services, Munich, Germany; and Sernova Corp., London, Ontario, Canada. For more information, visit www.hemacure.eu
ABOUT SERNOVA'S CELL POUCH SYSTEM
The Cell Pouch, as part of the Cell Pouch System, is a novel, proprietary, scalable, implantable macro- encapsulation device solution designed for the long-term survival and function of therapeutic cells. The device upon implantation is designed to incorporate with tissue, forming highly vascularized tissue chambers for the transplantation and function of therapeutic cells, which then release proteins and hormones as required to treat disease. The Cell Pouch, along with therapeutic cells, has been shown to provide long-term safety and efficacy in small and large animal models of diabetes and has been proven to provide a biologically compatible environment for insulin-producing cells in humans.
ABOUT SERNOVA CORP.
Sernova Corp. is developing regenerative medicine cell therapy therapeutic technologies using a medical device and immune protected therapeutic cells (i.e., human donor cells, corrected human cells and stem cell-derived cells) to improve the treatment and quality of life of people with chronic metabolic diseases such as insulin-dependent diabetes, blood disorders including hemophilia, and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. For more information, please visit www.sernova.com.
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