By William Swaney, Expression Therapeutics
Cell and gene therapy (CGT) companies transitioning from pure R&D and early preclinical work into clinical-phase development face several critical challenges. Key among them is the decision whether to outsource GMP manufacturing or to invest time, capital, and personnel toward development of in-house manufacturing capabilities.
It is vital to recognize each CGT program as unique and to carefully consider the inherent product needs — not only in early discovery but, more optimistically, through clinical testing and commercialization. An organization with high expectations for its product candidates and platforms will find it prudent to develop a customized process development plan for each product. That plan should include scale-up, but balance it against competing interests, such as time to clinical trial, phase of development, amount of product required per dose, need for single or repeat dosing, and justification of urgency (i.e., unmet medical needs where no suitable alternative treatments are available).
Due to the inherent complexity of cell and gene therapy products and manufacturing processes, it is crucial for sponsors to conduct due diligence before committing to a partner, be it a therapeutics company, a CDMO, or a supplier. Obviously, shared corporate culture, vision, and mission are important parameters for evaluating partners. Robust and scalable platform technology approaches that provide seamless transition from scaled-down bench research to pilot GMP applications, through to large-scale commercial manufacture, are advantageous. The ability of the technology to interface with the eQMS, EBR, and ERP systems is desirable. And both partners need to have the depth, resources, and a record of success to attract each other and support relationship longevity.