On January 10th, the British Medicine and Healthcare Regulatory Agency (MHRA) approved Genethon’s Phase I / II gene therapy clinical trial application in X-linked chronic granulomatous disease (XCGD) to start at Great Ormond Street Hospital in London. Genethon is also seeking approval for this multicenter trial in Germany, Switzerland and France. In total the trial will include 20 patients (5 per site) to be treated and followed up for 2 years. Genethon also obtained Orphan Drug designation for this treatment from the European Medicines Agency (EMA).
Read the press release – 5/2/2013 (2 pages – 41 ko)