Gene therapy Clinical trial commencement!

Our lab has commenced a gene therapy clinical trial, making us 1 of only 4 centres in Europe to carry out this trial! The clinical trial, sponsored by the US Pharmaceutical Company Biogen, targets an abnormal expansion of the MND-associated gene C90rf72 using a novel gene therapy that is designed to switch off the abnormal part of the gene. Up to 10% of all people diagnosed in Ireland with MND carry the abnormal C9orf72 gene, which can cause both MND and dementia. The Phase 1 trial which has been divided into 5 sections involving 75 patients worldwide, was recently approved by the HPRA (Health Products Regulatory Authority). Only 24 patients in the world will be enrolled in the fifth section, including the first Irish patient to enter the study. Best of luck to our team and all patients involved in the trial!!