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Gene Therapy Clinical Trial Reports Data – Including 3 Irish Participants

In March of this year, it was announced that the first Irish person with haemophilia had been treated with Gene Therapy for FIX deficiency. A further two people with haemophilia were then treated here in the same clinical trial.

The three are among 54 patients worldwide who participated in the clinical trial for severe haemophilia that started earlier this year. The positive early results of the trial were presented at the prestigious annual meeting of the American Society of Haematology (ASH) on Tuesday.

The Gene Therapy – called Etranacogene Dezaparvovec or AMT-061 uses a more active mutation of the FIX gene called the Padua mutation to give higher FIX expression levels. At the ASH Conference, the Phase 3 clinical trial results were discussed at the point where 6 months post treatment data was available on all 54 people who participated. Of the 54, 98% achieved FIX levels of more than 5%, 83% achieved FIX levels greater than 20%, 68% achieved levels greater than 30% and 38% achieved levels greater than 40% at 6 months post Gene Therapy infusion

The three Irish patients were dosed in February and March this year at the Clinical Research Facility at St James’s Hospital and have been monitored closely since then. All 3 Irish patients have achieved very good levels of FIX expression to date.

One of the three Irish patients is Irish Haemophilia Society CEO Brian O’Mahony, who describes himself as “functionally cured”. Brian was interviewed by RTE & Newstalk this week, both of which make for very interesting listening.

You can also read the Irish Times article here.

Listen on Newstalk website

Listen on RTE website