The Logistics of Developing an Emerging Therapy

Developing emerging modalities such as cell and gene therapies presents many challenges from selecting the right approach to overcoming manufacturing hurdles. Cell therapies can be personalized/patient-derived (autologous) or produced from donor cells (allogeneic). Each has its own issues, notably logistics and quality for the former and heterogeneity and immune rejection for the latter. The approach, indication, required dose, and patient population all impact the overall cost of each type.

The biggest challenges for gene therapies, most of which are delivered using viral vectors, are related to manufacturing issues around scalability, productivity, and overall recovery and limited availability of plasmid DNA, a key raw material for viral vector production via transient transfection. Manufacturing requirements are, in fact, more complex that those for protein- and antibody-based therapeutics. Many contract service providers are, fortunately, investing int eh development of optimized, flexible manufacturing platforms and stable producer cell lines to help overcome these issues.