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4 BioPharm International ® Quality and Regulatory Sourcebook eBook March 2024 www.biopharminternational.com C ell and gene therapies (CGTs) offer the poten- tial to revolutionize healthcare by addressing unmet medical needs. However, the science is advancing at a pace faster than regulatory authorities can issue relevant laws, regulations, and guidance documents. Therefore, it is imperative that the industry apply appropriate compliance controls in the manufacture of CGT products. These products pose unique manufacturing and compliance chal- lenges due to their inherent nature (i.e., originating from living organisms, being used in the treatment of only one or a limited number of persons, extreme- ly small batch sizes, and challenging contamination control). There is limited manufacturing and regula- tory experience with CGTs, as only few products have yet obtained marketing authorization; 31 CGTs were approved by FDA (1) by mid-2023 and 25 by the Euro- pean Medicines Agency (EMA) (2). CGTs are termed ad- vanced therapy medicinal products (ATMP) in Europe. Yet, the pipeline is still growing, with more than 2000 CGTs/ATMPs in clinical pipelines (3). These prod- ucts are at the forefront of research and, as a conse- quence, many companies involved in their discovery are small and medium-sized enterprises, often with staff who are inexperienced or unfamiliar with reg- ulatory expectations and/or spin-offs from academic research centers. The importance of applying the appropriate level of compliance in the respective phases of the product's lifecycle cannot be over-emphasized. The chemistry, manufacturing, and controls (CMC) for these prod- ucts will be scrutinized by the approving regulatory authorities, such as FDA and EMA. Any delay to the approval of clinical trials or license to market the CGT will not only be costly, but it will also delay essen- tial therapies, often without any alternative, for pa- tients. For instance, some sponsors have experienced delays with FDA at key trial milestones for reasons Phase-appropriate Compliance for Cell and Gene Therapies Zhongren Wu, PhD, is principal consultant at Parexel; Siegfried Schmitt, PhD, is vice president, Technical, at Parexel; and Lynne Ensor, PhD, is senior director, Pharma Biotech and Dietary Supplements Consulting at NSF. Understanding how to apply phase-appropriate GMPs is crucial for achieving successful regulatory approval. THONGDEN_STUDIO - STOCK.ADOBE.COM