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30 Pharmaceutical Technology BIOLOGICS AND STERILE DRUG MANUFACTURING 2019 P h a r mTe c h . c o m Manufacturing W hile gene and cell therapies have been touted as the future of medicine for decades, there is evi- dence to indicate that they are finally poised to deliver results. Several products are already on the market, including Kymriah, Yescarta, and Luxturna, and many others are advancing to late-stage clinical development and commercialization. A number of different manufacturing platforms are being developed to manufacture both autologous and allogeneic therapies. In the United States alone, there are 34 gene therapies in pivotal trials and another 470 in earlier stages of clinical testing (1). Although the long-term transformative promise of gene and cell therapies is becoming increasingly clear and is good news for many patients, these treatments also present unique chal- lenges for a number of stakeholders. Factors that drug develop- ers, regulators, investors, and others must consider include the fact that these therapies often target very small patient popula- tions; have shorter treatment windows; offer potentially cura- tive efficacy; have high up-front costs; lack long-term efficacy and safety data; and involve complex, expensive, and high-risk manufacturing processes. Challenges to commercialization Each of these factors can have a significant impact on the clinical development and regulatory review process and on the chance of successful commercialization. For teams involved in investment and planning related to technology and manufacturing, it is essential to The Link Between Manufacturing and Commercialization in Gene and Cell Therapy The highly customized nature of cell and gene therapy production means that manufacturing innovations for one therapy may not be easily transferable to others. Walter Colasante, Pascale Diesel, and Lev Gerlovin are vice-presidents in Charles River Associates' Life Sciences Practice. The authors wish to acknowledge the contributions of Stephanie Donahue and Michael Krepps to this article. The views expressed herein are the authors' and not those of Charles River Associates or any of the organizations with which the authors are affiliated. SCIENCE PHOTO/STOCK.ADOBE.COM Walter Colasante, Pascale Diesel, and Lev Gerlovin