The TREAT-NMD Alliance held its 5th international conference focused on translational medicine in inherited neuromuscular diseases at the end of November 2017 in Freiburg, Germany. Genea Biocells was proud to support this meeting which attracted over 200 delegates including academics, industry professionals, patients and caregivers, patient advocacy organisations and clinical specialists. It was a great opportunity for us to meet colleagues and collaborators and to network on neuromuscular disease drug development.
Scientifically, the conference provided the latest updates on standards of care, outcome measures and patient registries. Of particular interest to us was the session “emerging strategies for NMD treatment” which kicked off with a talk by John Porter from the Myotonic Dystrophy Foundation (USA) with an excellent overview on the drug discovery process and a word of caution not to over-interpret data from animal studies: mice are not human and do not develop human disease. While they are useful models, mice or other animals are still simply an experiment and do not indicate a “cure”. Ana Buj Bello (Genethon, France) gave an update on an AAV-based gene therapy approach for X-linked myotubular myopathy, a severe disease affecting about 1:50,000 males with a median life expectancy of 2 years. After studying mouse and dog models it was great to hear that a human vector was developed and approved by the FDA in 2017. A clinical trial commenced last September giving hope to affected kids and their parents. Other speakers covered a range of diseases, including myotonic dystrophy type 1 (Charles Thornton, University of Rochester, USA) and LAMA2 (Markus Ruegg, University of Basel, Switzerland).
Genea Biocells’ Director of Business Development, Colin Tristram, and President Uli Schmidt enjoyed discussing our progress in drug development for facioscapulohumeral muscular dystrophy (FSHD) and spinal muscular atrophy (SMA) with scientists, clinicians and particularly patients attending the conference. Another highlight was Freiburg’s historic Christmas market with its many “Gluhwein” stalls which more than compensated for the very cold and wintery weather.