Engineering the next generation of cell-based therapeutics

Cell-based therapeutics are an emerging modality with the potential to treat many currently intractable diseases through uniquely powerful modes of action. Despite notable recent clinical and commercial successes, cell-based therapies continue to face numerous challenges that limit their widespread translation and commercialization, including identification of the appropriate cell source, generation of a sufficiently viable, potent and safe product that meets patient- and disease-specific needs, and the development of scalable manufacturing processes. These hurdles are being addressed through the use of cutting-edge basic research driven by next-generation engineering approaches, including genome and epigenome editing, synthetic biology and the use of biomaterials. The widespread clinical translation and commercialization of cell-based therapies are hampered by challenges related to cell source, viability, potency, safety and scalability. Here, Veiseh and colleagues overview progress in the development of cell-based therapeutics and discuss how biological engineering approaches — including genome editing, synthetic biology and the use of biomaterials — are beginning to address key challenges in the field.