Cell Therapy – Falling short of its potential?
Despite major investment in the field, few cell therapies have gained regulatory approval with limited commercial success. The success of cell therapy products is defined by three factors: safety, efficacy and scalability. Achieving these for cell therapy presents new challenges; overcoming transplant rejection (and GvHD in the case of immune cells), producing and culturing functional cells and developing an off-the-shelf therapy with a scalable manufacturing process. Gene editing technologies (CRISPR, TALENs and ZFNs) are paving the way towards allogeneic safety and ‘universal donor cells’. However, progress is difficult without the ability to produce functional cells at scale. A bioinformatics approach to systematically control the transcriptomic network with transcription factors and/or small molecules to culture or convert any cell type opens up the opportunity to develop any autologous, allogeneic cell therapies and a new class of therapies: in vivo reprogramming.View all resources