New €5.6 million project aims to develop cell-based haemophilia A therapy

Jan 25 2016

Loughborough University is working with a team of international partners on a new €5.6 million project to develop a cell based haemophilia A therapy.

People with haemophilia have prolonged abnormal bleeding as a result of trauma. Haemophilia A, also called factor VIII (FVIII) deficiency, is the most common form of the genetic disorder and is caused by the missing or defective blood clotting protein FVIII.

The HemAcure consortium, with coordinating institute, University Hospital Würzburg has received funding from the European Commission's Horizon 2020 programme to try and significantly improve the quality of life of people suffering from haemophilia A.

The clinical product being developed by the consortium will use the patient's own cells taken from a blood sample and corrected for the FVIII gene.  These cells, placed in an implanted pre-vascularized GMP contract manufactured Cell Pouch™ device, will release FVIII on a continual basis at a rate that would be expected to significantly reduce disease-associated haemorrhaging and joint damage - a common problem in people with haemophilia.

The constant delivery of FVIII is also expected to reduce or eliminate the need for multiple weekly infusions, which is the current standard of care.

Loughborough's involvement in the project is being led by Dr Alexandra Stolzing from the University's Centre for Biological Engineering in the School of Mechanical and Manufacturing Engineering. She will be responsible for upscaling cell production, ageing biomarkers and cell safety testing.

Dr Stolzing, a Senior Lecturer in Regenerative Medicine, said: "We are delighted to be a part of this ground breaking project.  Not only could it make a significant difference to the lives of thousands of people, it will also dramatically reduce the financial pressures on patients and healthcare providers."