Quality System Overview For GMP Manufacturing

When you think about cell and gene therapies (CGTs), what most often comes to mind is the early stage scientific innovation responsible for finding new ways to treat diseases. A lot of complex research and testing is performed to confirm proof of concept and that the therapy performs as desired. That is a huge accomplishment for most CGT companies. The next step is to prepare the product for in-human clinical testing. Clinical protocols are developed in consultation with the regulatory authorities, a target patient population is identified, clinical investigators and an appropriate contract research organization (s) are selected, and funds are obtained by the CGT company to begin the clinical studies. Again, this is a huge milestone for CGT companies. Unfortunately, what is sometimes overlooked or left to the last minute is the less-than-glamourous, but still critical, manufacturing steps that ensure a new, potentially lifesaving treatment reaches patients in a safe and reliable form.

At CCRM, we are often asked by CGT companies to help them understand and implement Good Manufacturing Practices (GMP) for their clinical trial materials; therefore, we created this overview of the mandatory quality systems and functions that are required within a standard GMP facility to comply with ICH Guidelines including Q7 and EU ATMP guidelines, Canadian GMP (C.002) and U.S. FDA, PHS Act section 351/361 and CFR 21 Parts 210/211 and Part 11 as they apply to cell and gene therapy. Other guidelines are considered, as required, such as ISO 9001:2015 certification.