European Commission Grants $6M to Develop New Hemophilia A Treatment

Note: The following is an edited press release from Sernova Corp. The original release can be read in its entirety here.

Sernova Corp., a clinical stage regenerative medicine company, announced on Monday, December 21, 2015, that the European Commission’s Horizon 2020 program has awarded a Euro 5.6M ($6.1M USD) grant to a consortium consisting of Sernova Corp and five European academic and private partners to advance development of a GMP clinical grade Factor VIII releasing therapeutic cell product in combination with Sernova’s Cell Pouch(TM) for the treatment of severe hemophilia A, a serious genetic bleeding disorder caused by missing or defective factor VIII in the blood stream.
The current market for factor VIII is US$5.0B/year, with a cost of up to US$260,000 per patient each year. Current standard of care involves regular infusions of factor VIII, which achieves normal factor VIII blood levels for only a few hours at a time. The product being developed by the HemAcure consortium will seek to provide constant delivery of Factor VIII to normalize blood levels in an effort to significantly improve the quality of life of patients suffering from hemophilia A.
The product being developed by the HemAcure consortium is expected to be highly disruptive to the current standard of care treatments for hemophilia A. The therapeutic goal of the product is to use the patient’s own cells corrected for the factor VIII gene. These 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.
“The therapeutic potential to have a constant release of factor VIII from a hemophilia A patient’s own genetically corrected cells placed within the implanted Cell Pouch would be a very significant advance in the treatment of hemophilia A,” remarked Dr. David Lillicrap, MD, FRCPC Professor Department of Pathology and Molecular Medicine Queens University, Canada Research Chair in Molecular Hemostasis and member of the HemAcure Scientific Advisory Board. “Sernova’s Cell Pouch with its vascularized tissue lined chambers for therapeutic cells, which has already been proven for islet safety and survival in human clinical assessment of diabetes, is an ideal, fully scalable first-in-class medical device suitable for the potential treatment of hemophilia,” added Dr. Lillicrap.
The preliminary preclinical proof of concept data used as a basis to support the foundation of the grant was generated in a collaborative agreement between Medicyte GmbH under the FP7 ReLiver project, grant agreement 304961 and Sernova Corp where cryopreserved cells with the ex vivo inserted corrected gene for factor VIII were successfully shipped and assessed in Sernova’s Cell Pouch at its headquarters in Canada. Regarding Sernova’s participation in the consortium, the review of the HemAcure grant proposal stated the following, “Participation of the third country partner (Sernova Corp) is essential for carrying out the (program). This is justified by the fact that the partner in question is the one who possesses the technology that will be the basis of the whole proposal, and which will perform all the in vivo studies. Sernova uses a scalable, contract manufactured, proprietary patented worldwide implantable medical device (Cell Pouch) transplanted with therapeutic cells. (It) has been in development for more than six years and has shown success in multiple small and large animal preclinical models and is now in a clinical trial for another therapeutic indication. This Cell Pouch device is the only such device that when implanted under the skin is proven to become incorporated with blood vessel enriched tissue-forming chambers for the placement of therapeutic cells. This implies that the Canadian partner (Sernova) is an essential partner for the success of this project.”