James Warren, PhD, Vice President, Pharmaceutical Development; Leader, Biotechnology and Gene Therapy Development, Ultragenyx Pharmaceutical
In the past few years, several cell and gene therapy products have gained regulatory approval in the US and EU with many more in the pipeline. Manufacturers of gene therapy products must tackle technological challenges under the pressure of short timelines resulting from streamlined clinical development. This presentation will focus on the key technical development challenges facing the industry as product development programs move into the later stages of process development and scale-up, process performance qualification, and ultimately, commercialization.