De-risking pluripotent stem cell therapies

The traditional development of pluripotent stem cell therapies is fraught with risks that often prevent successful commercialization. The interest in advanced therapies produced from pluripotent stem cells is growing rapidly, with the number of clinical studies across the globe increasing from just 11 in 2015 to more than 100 in 2024 [1][2]. The global commercial market for induced pluripotent stem cells is expected to grow from US $3.4 billion in 2023 and forecast to reach $5.2 billion by the end of 2028, at a compound annual growth rate of 9.1% from 2023 through 2028 [3]. In this discussion, we delve into various challenges of the development journey, from sourcing suitable cell lines in compliance with cGMP to handling the diversity in cell differentiation efficiencies, the labor-intensive nature of traditional manufacturing and poorly controlled processes, and the relevance of strong nonclinical data packages. It highlights the need for a shift from reactive to proactive risk mitigation strategies to enhance program success. Additionally, the article suggests a holistic approach to development, focusing on a deep understanding of the final commercial product, early risk assessment with the ‘end goal’ in mind, maintaining simpler manufacturing processes, and engaging with regulators early to ensure a smoother path to commercialization.

Please enter your email to access content Read now