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1st person with haemophilia in Ireland to be treated with ground-breaking gene therapy

The Wellcome-HRB Clinical Research facility in St James’s Hospital has announced it is participating in a ground-breaking Gene therapy trial. This is first time this revolutionary therapy has been trialled to treat a person with haemophilia in Ireland. The principal investigator of the study is Dr. Niamh O Connell from the National Coagulation Centre in St. James’s Hospital.

Standard treatment since the 1970’s has been intravenous infusions of the missing clotting factor but several companies and academic institutions have been working for many years on innovative gene therapy treatments for haemophilia.

The Irish recipient of the gene therapy, infused at the Clinical Research facility in Dublin, is part of a Phase 3 clinical trial. The therapy uses an adeno-associated viral vector to deliver the Factor IX gene therapy intravenously to the liver of the individual who has severe Haemophilia B. In the earlier Phase 2B trial of this particular gene therapy, the Factor IX level in the blood increased from less than 1% to between 33% and 51% in the small number of individuals treated. This transforms their quality of life from having severe haemophilia to mild haemophilia or no haemophilia.

Speaking about the development, Professor Martina Hennessy, Director of the Clinical Research facility and Associate Professor in Trinity’s School of Medicine said:” Access to high quality research is an integral part of good healthcare because it raises standards and pushes the boundaries of what can be achieved. Delivering gene therapy requires specialised training and equipment, we have been preparing with Dr O’Connell and her team for over a year to undertake this exciting work, in partnership with the Irish Haemophilia Society. Other trials are planned, we hope this expertise leads other Irish patient groups also being able to access potentially life changing treatments in the future.”

Professor Martina Hennessy
Director of the Clinical Researc
h facility and Associate Professor in Trinity’s School of Medicine.