Gene and cell therapies promise game changing therapeutic outcomes but they also present unique challenges both before the clinic and during the clinical development phase. Before the clinic it is important to establish an appropriate manufacturing capability and manufacturing process for the investigational product. It is equally important to develop a nonclinical toxicology strategy that will not only satisfy regulatory expectations but is also practical in terms of time and resources required.
Drawing on the experience of the Novotech group in bringing gene and cell-based therapies into and through clinical trials, a range of clinical development case studies will be presented focusing on development performed in Australia.