The past decade has seen a shift in drug discovery to include targeting the root cause of genetic diseases: loss-of-function, gain-of-function, and change-of-function mutations in the genome. Although first generation genetic medicines like gene therapies heralded a new approach to treating previously untreatable diseases, the clinical application of the drugs was fraught with obstacles that involved restricted biodistribution, low tolerability, and limited durability, in addition to processing problems such as manufacturability and scalability. A new generation of scientists and companies have learnt key lessons from these first-generation genetic medicines and developed tools and techniques to circumvent the obstacles they faced. This has resulted in a new class of genetic medicines.
In this GEN Protocols’ Expert Exchange, Dietrich Stephan, PhD, Chairman and CEO of NeuBase, talks about the tools and technologies that his company is using to develop the next generation of genetic medicines that incorporate strategic lessons learnt from first generation drugs that pioneered targeting the genome. Dietrich Stephan, PhD, is CEO at Neubase Therapeutics, a Pittsburgh biotech developing a novel class of precision genetic medicines to address genetic diseases at the base level. Dietrich has helped launch 14 biotechnology companies to advance novel molecular diagnostics, and was a co-founder of Navigenics, a pioneering consumer genetics company. Neubase is targeting several neurological disorders, including Huntington’s disease and myotonic dystrophy.