Audentes customer case with Biotage PhyPrep system

Gene therapy has had its share of setbacks, but finally gene therapies are proving successful at treating diseases like neuromuscular disorders, cancer and blindness. Adeno-associated virus (AAV) has emerged as a leading gene delivery vehicle because it is efficient and has demonstrated safety in humans. Although obstacles remain, optimism about AAV gene therapy has spurred an influx of potential gene therapies into the research pipeline. Biotage is helping to speed up the flow of research with automated systems such as their PhyPrep plasmid purification system.

Mark Champe manages a gene therapy research lab in South SanFrancisco, previously called Audentes, which is now part of the Japanese pharmaceutical company Astellas Pharma. Before being acquired last year, Audentes Therapeutics had developed a gene replacement therapy for X-linked myotubular myopathy. X-linked
myotubular myopathy is a devastating disease, causing extreme muscle weakness and respiratory failure. It is lethal in half the boys who have it and the other half will require a ventilator tobreath and a wheelchair for mobility.