The CureCN European trial aims to assess the product’s safety, identify the optimal dose, and evaluate the therapeutic efficacy of the drug candidate. The trial is now at a very important stage, the end of the second cohort and presentation of first results at the International Liver Congress, which took place from 23-26 June, 2021.
In that context, the French Crigler-Najjar Patient Association (AFCN) organized a meeting in visio (due to the pandemic situation) and the Italian Patient Association (CIAMI) organized an in-person meeting near Bologna. Both associations gave updates on the trial and the meeting also served to allow patients and families to have the opportunity to discuss with scientific and medical experts engaged in the project.
During the AFCN meeting, participants had the opportunity to talk to Giuseppe Ronzitti and Fanny Collaud from Genethon and Philippe Labrune from Hôpital Béclère. Dr Lorenzo D’Antiga from Azienda Ospedaliera Papa Giovanni XXIII attended the meeting of the Italian group.
Both meetings illustrated the excellent results of the ongoing gene therapy and the trial's next steps. Families and patients had the opportunity to address their questions. In France, many questions, in particular, were asked about concerns regarding the elimination of antibodies. Patients and families in Italy had the opportunity to talk to patients that are included in the trial. Furthermore, there was great interest about the ‘after trial’ period, which would include commercialization of the product and access to the treatment for all CN patients.
The promising results of the trial increase the hope of the CN patients to be closer to a cure. They are grateful to all the partners of the consortium for the work accomplished and are looking forward to seeing how the trial and the work of the consortium continues.