Non-viral in vivo cell and gene therapies: A change journey for CMC science and scientists

The CMC (Chemistry, Manufacturing and Controls) landscape is rapidly evolving as pharmaceutical science shifts from traditional small molecules and peptides towards non-viral in vivo cell and gene therapies. The article highlights the skills required including hybrid scientific roles spanning chemistry, biology, drug delivery, formulation and analytical development. These are essential to deliver clinical non-viral in vivo cell and gene therapy products. In addition, organizational structures that are best suited for these new therapies are discussed. To frame the discussion on skills, we briefly start out by summarizing the increased modality complexity in terms of multicomponent LNPs (lipid nanoparticles), including novel excipients, new product manufacturing methods, complex analytical requirements, and a regulatory framework that is still developing. Finally, it is highlighted how non-viral in vivo cell and gene therapies will have clinical and patient access advantages. We hope to provide inspiration for scientists on a similar journey and demonstrate that expanding your skills, knowledge and science is a worthwhile undertaking to aid in developing the differentiated medicines of the future.