Turning Genes into Medicines: Gene Therapy for Genetic Disease

One of the goals of the Human Genome Project was to improve therapeutic options for those born with serious inherited diseases. Today gene therapy is beginning to realize this promise, with the ever-increasing level of clinical investigation in the space, and the first products coming to licensing in the last few years. In this podcast, first recorded at the 2019 PODD: Partnership Opportunities in Drug Delivery conference, Dr Katherine High, Spark Therapeutics, reviews the current state of gene therapy, including the development of therapies for diseases that were previously untreatable, and, for diseases where therapeutics already existed, the development of therapies that greatly lessen the burden of treatment for the patient and the family. Hurdles in clinical development of gene therapy will be discussed. Examples will be drawn from already approved products and from investigational agents in late stage development. To learn more about the 10th annual PODD: Partnership Opportunities in Drug Delivery conference please visit www.theconferenceforum.org