The European Commission will award the Brussels-based HemAcure Consortium €5.6 million in next-stage funding of a therapy called Cell Pouch — developed in collaboration with Canada’s Sernova — for hemophilia A patients.
The therapy delivers corrected factor VIII with the help of cells that sit in a transplanted pouch under the skin. The cell pouch itself is already in clinical trials in patients with diabetes, where insulin-producing cells are used instead of those making factor VIII.
“The international HemAcure Consortium team members are pleased with the groundbreaking scientific advances achieved at this point and are on track for this regenerative medicine solution to advance into human clinical evaluation,” Dr. Philip Toleikis, president and CEO of Sernova, said in a press release.
Before the therapy can enter human trials, researchers must test its safety and efficacy of the cell therapy in animals. The money, which is part of the EU Horizon 2020 Grant Award, will support further studies.
So far, researchers have developed a method for gathering endothelial cells from patients’ blood and correct the faulty factor VIII gene. Endothelial cells line the inside of blood vessels and produce the clotting factor in healthy people. Once the cells are corrected, the idea is to let them expand before being transplanted back into the patient inside the cell pouch.
The HemAcure Consortium comprises Sernova, a quality management firm and three European academic institutions.
“Sernova’s Cell Pouch platform technologies are achieving important world 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,” said Toleikis.
Added Dr. Joris Braspenning, HemAcure’s program coordinator: “We are thrilled with the EU’s approval of the next stage of funding based on our quality interim report. This is a strong validation of the consortium’s dedication and teamwork and the importance of this regenerative medicine approach.”