Formulation and Quality – Biomolecular
2019 PharmSci 360
Cell and gene therapies hold great promise for several unmet medical needs, as seen by recent FDA and EMEA approvals of CAR T cell therapies for oncology purposes and ex vivo gene editing to cure hereditary blindness. Many of these therapies are ex vivo autologous approaches, at a high level involving removing cells from the patient, manipulating them to express the desired features, and reinstating them back into the patient for the therapeutic benefit. While promising from a healthcare perspective the therapies present difficulties from a CMC perspective in terms of development and manufacturing of reagents and the final cell product, but also quality control aspects as analytical assays are very different than those used for more traditional modalities. Finally, the regulatory landscape is still developing and so are guidance for global distribution. This presentation will give an overview over CMC issues along with opportunities for solutions in this developing field.