Ground-breaking gene therapy used for first time in Ireland to treat haemophilia

Ground-breaking gene therapy has been used for the first time to treat a person with severe haemophilia in Ireland.

Ground-breaking gene therapy used for first time in Ireland to treat haemophilia

Ground-breaking gene therapy has been used for the first time to treat a person with severe haemophilia in Ireland.

With advances in gene therapy, it is hoped that haemophilia, an inherited bleeding disorder, will no longer be a lifelong disease.

The Irish Haemophilia Society said the recipient, who wants to remain anonymous, received the therapy as part of a clinical trial.

There is currently no cure for haemophilia but patients can be treated with an intravenous clotting factor.

Gene therapy provides patients with haemophilia a new “working copy” of the genes for clotting factor protein.

The Irish person who has severe Haemophilia B received the Factor IX gene therapy intravenously to the liver and it started to produce the missing clotting factor.

Irish Haemophilia Society chief executive Brian O’Mahony said: “This is a momentous occasion for the haemophilia community in Ireland”.

“In the past, viruses such as HIV and Hepatitis C decimated the haemophilia population in Ireland through contaminated blood,” said Mr O’’Mahony.

“It is ironic that a virus could now be the delivery system which offers the best hope of a practical cure for severe haemophilia” he stated.

Haemophilia gene therapy trials use a virus called adeno-associated virus (AAV) to get the gene into the body.

AAV are small viruses that are not currently known to cause disease but can cause a very mild immune response.

The gene therapy used in the trial has been found to increase the Factor IX level in the blood from less than 1% to between 33% and 51% in a small number of individuals treated.

This transforms their quality of life from having severe haemophilia to mild haemophilia or no haemophilia.

“It is hoped that the effect of the gene therapy infusion will last for many years and possibly for a lifetime,” Mr O’Mahony said.

Dr Niamh O’’Connell from the National Coagulation Centre in St James’’s Hospital said the gene therapy was one of several active trials in novel therapies for haemophilia in Ireland.

The gene therapy was infused in the Clinical Research facility in St James’s Hospital that is co-funded by the Wellcome Foundation and the Health Research Board. Its director, Prof Martina Hennessy, said other trials were planned.

“We hope this expertise also leads to other Irish patient groups being able to access potentially life-changing treatments in the future.”

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