CASSS Welcome & CGTP Summit 2024 Introduction

Advancing Comparability Understanding of Cell-based Medicinal Products

Despite early success of adoptive cell therapies  and AAV-based gene therapies, it is quite evident that bringing such advanced medicines to the market has proven to be significantly challenging, most importantly due to CMC concerns. Amongst many challenges faced by CGT developers, comparability has continuously emerged as a recurring and inevitable hurdle due to the poorly understood and characterized nature of CGT products. During the first edition of the CGTP Summit in 2023, comparability considerations and current industry practices were discussed focusing on AAV-based gene therapies and CAR T-cell therapies. Reflecting on past success, the summit will continue the discussions on comparability with a focus on cell-based therapies.    

Cell-based medicinal products are inherently one of the most complex therapeutic modalities developed so far. Due to live cells   and inherent variability from where they are sourced and stage of development, cell-based medicinal products have proven to be difficult to characterize and standardize with currently available analytical methodologies. This summit will showcase presentations from the CGT industry and hold interactive panel discussions to tackle some of the challenges seen with conventional comparability strategies for cell-based products.   In this summit, we will focus on somatic cell products and cell-based gene therapy products.

Finally, this summit will feature an expert regulatory panel comprised of regulators from key regulatory agencies. To that effect, this summit will discuss the recently issued FDA draft guidance on manufacturing changes and comparability for human cell and gene therapy products. The purpose of this panel is to openly discuss the common comparability challenges, to engage in a productive dialogue about mitigating those challenges and opportunities for harmonization between regulatory agencies.