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Implantable cell-based device could offer treatment for hemophilia.

A new $8.5 million (CAD) research grant award by the European commission via its Horizon 2020 program aims to address patients affected by hemophilia. The funds are earmarked to the HemAcure consortium with the goal to bring treatment of hemophilia A, also called factor VIII deficiency--the most common form of hemophilia A--into the clinic for human trials. The consortium, consisting of Canadian-based Sernova Corp. and five European academic and private partners, will be working jointly to advance development of a GMP clinical grade Factor VIII releasing therapeutic cell product via Sernova's signature technology called the Cell Pouch.

The therapy being developed by the HemAcure consortium is expected to be highly disruptive to the current standard of care treatments for hemophilia A. The therapeutic uses the patient's own cells that have been corrected for the factor VIII gene. Central to the therapy is Sernova's Cell Pouch System, an implantable and scalable medical device that 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.

The patient's corrected cells placed in the implanted Cell Pouch will release factor VIII on a continual basis at a rate that would be expected to significantly reduce disease-associated hemorrhaging and joint damage. The constant delivery of factor VIII is also expected to reduce or eliminate the need for multiple weekly infusions, which is the current standard of care using plasma-derived or recombinant, genetically engineered factor VIII for the prophylactic treatment of hemophilia A.

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Title Annotation:In Development
Publication:Medical Product Outsourcing
Date:Jan 1, 2016
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