Exploring the Association Between Cell and Gene Therapy Product Development and Pharmaceutical Company Features

Abstract Background The number of pharmaceutical companies developing cell and gene therapy (CGT) products, categorized into cell therapy products (CTPs) and gene therapy products (GTPs), has increased, expanding patient access to approved treatments. Compared to conventional modalities, such as chemical compounds and biopharmaceuticals, both CTPs and GTPs pose significant challenges in their development. This study analyzed the characteristics of pharmaceutical companies associated with entry into CGT product development. Methods Focusing on 309 pharmaceutical companies worldwide with marketed products, we analyzed entry into development and the numbers of CTPs and GTPs. We explored firm characteristics associated with entry into CTP and GTP development, as well as their respective proportions within company pipelines, using multiple regression models. Results Approximately 30% of firms included CTPs or GTPs in their product baskets. Larger firms were more inclined to pursue CGT product development. Compared with companies headquartered elsewhere, Japanese companies were more likely to develop CTPs, maintaining a higher proportion of these agents in their baskets. The tendency towards home market entry was more pronounced for CTPs and GTPs than for chemical moieties and proteins. Firms with experience in biopharmaceuticals were more likely to develop GTPs, demonstrating higher proportions of these agents than firms without such experience. While larger sales tended to enhance entry into both CTP and GTP development, research and development expenditure played a greater role in driving entry into CTP development than into GTP development. Conclusions These findings underscore the distinct characteristics of CTPs and GTPs and offer valuable insights into pharmaceutical innovation research.