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Breakthrough in regen therapy for Hemophilia A: HemAcure
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consortium made up of several European universities and an Ontariobased biotech company reported they have achieved several breakthroughs in their work on a regenerative medicine therapy to treat Hemophilia A. People with Hemophilia have prolonged abnormal bleeding as a result of trauma. Hemophilia A, also called Factor VIII (FVIII) deficiency is the most common form of Hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe Hemophilia occurs in about 60 per cent of cases where the deficiency of FVIII is less than one per cent of normal blood concentration. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene. The therapy being developed by international scientific HemAcure Consortium is to treat severe Hemophilia A, with Sernova Corp.’s implanted Cell Pouch device transplanted with therapeutic cells. The cells are corrected to produce Factor VIII at a level sufficient to significantly reduce the side effects of the disease and improve patient quality of life. The Cell Pouch system is a novel implantable and scalable medical device. It forms a natural environment in the body for the housing and long-term survival and function of therapeutic cells. These therapeutic cells release necessary proteins or hormones missing from the body to treat chronic diseases as an alternative to daily administration of drugs. This project has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No. 667421. The EU Commission awarded the HemAcure consortium €5.6 million in next-stage funding of the Cell Pouch therapy. The consortium members include the University Hospital Wuerzburg (Coordinating Institute), Germany; IMS - Integrierte Management, Heppenheim, Germany; Università del Piemonte Orientale “Amedeo Avogadro,” Novara, Italy; Loughborough University, Loughborough, United Kingdom; ARTTIC 26 BIOTECHNOLOGY FOCUS August/September 2017
• In combination, this work is in preparation for safety and efficacy studies of human Hemophilia corrected Factor VIII producing cells in the Cell Pouch in a preclinical model of Hemophilia.
International Management Services, Munich, Germany; and Sernova Corp., London, Ontario. The following developments have been achieved by the consortium: • A reliable procedure has been implemented to isolate and maintain required endothelial cells from a sample of the patient’s blood. • Using a novel gene correction process, the cells have been corrected and tuned to reliably produce the required Factor VIII to treat Hemophilia A. • The cells have been successfully scaled up to achieve the required therapeutic number, and cryopreserved for shipping and future transplant into the implanted Cell Pouch. • A preliminary study confirmed survival of the Factor VIII corrected human cells injected into the Hemophilia model, achieving sustained therapeutic Factor VIII levels. This preliminary work is being used to aid in dosing of these cells in the Cell Pouch. • Safe Cell Pouch surgical implant and cell transplant procedures have been developed in the Hemophilia A model in preparation for use in Hemophilia patients. • Development of Cell Pouch vascularized tissue chambers suitable for Factor VIII producing cell transplant has been demonstrated in the Hemophilia A model, expected to mimic the predicted findings in human patients.
“The international HemAcure Consortium team members are pleased with the ground breaking scientific advances achieved at this point and are on track for this regenerative medicine solution to advance into human clinical evaluation,” remarked Dr. Philip Toleikis, Sernova president and CEO. “Sernova’s Cell Pouch platform technologies are achieving important world-first milestones in both diabetes and now Hemophilia, two significant clinical indications which are being disrupted by its regenerative medicine approach aimed at significantly improving patient quality of life.” “This is a strong validation of the Consortium’s dedication and teamwork and the importance of this regenerative medicine approach,” said Dr. Joris Braspenning, HemAcure program coordinator. With Factor VIII corrected cells, studies are ongoing to optimize cell dosing within the Cell Pouch and for the study of safety and efficacy of Hemophilia corrected Factor VIII cells in the Hemophilia model. These studies support the current extensive regulatory package already assembled for the Cell Pouch in anticipation of human clinical evaluation of the Cell Pouch with Hemophilia corrected Factor VIII producing cells. “…these fundamental advancements have set the stage for further optimization and implementation of cell production processes under controlled GMP conditions,” according to Martin Zierau, IMS member consortium team leader responsible for coordination of GMP processes.
To see this story online visit https://biotechnologyfocus.ca/breakthrougprogress-in-regen-therapy-for-hemophilia-ahemcure/
