Cell Therapies Pty Ltd hosted a very unique breakfast session at ISCT on Thursday 30th May. Our own Nathan Smith chaired this event, which gave a deep look into clinical development of a gene modified cellular therapy for a rare disease, being developed and tested in multiple countries. Mr Geoff MacKay, CEO of Avrobio, started us off with an in-depth presentation on Avrobio’s motivation and approach to treating rare lysosomal storage disorders with genetically modified cells, using Avrobio’s lentiviral-based technology. Then Dr Alexandra Rizzitelli, one of our fantastic senior project managers, presented the approach Cell Therapies and Avrobio have taken to transfer the cell and gene processes to our site in Australia, develop it for use in a clinical trial, and ultimately start treating patients in a Fabry Disease trial. Professor David Ritchie of the Royal Melbourne Hospital, then added the clinical dimension to the session, by giving a moving description of the impacts of Fabry Disease on patients and their families, and how the approach of using gene-modified cellular therapies is being tested in the clinic.
Overall this session was a great review of the multiple moving parts necessary to bring a unique and promising gene therapy into clinical trials. Thanks to all our speakers!