Set Your Cell And Gene Therapy Program Up For Success From Day One

Due to the complexity and innovative nature of cell and gene therapies (CGTs), their discovery and initial development often occur in academic labs, translational institutes, or small biotech startups. While these entities excel in scientific innovation, they may lack expertise in later-stage development and commercialization. The development of CGTs already comes with high costs, even when everything runs smoothly, and any missteps can lead to increased expenses, delays, or other issues that may affect the therapy's success further down the line. It is essential for early-phase researchers to understand how their decisions impact the program's long-term prospects, particularly with respect to regulatory approval, costs, and timelines.

Academic institutions, translational centers, and biotech startups frequently struggle with meeting regulatory requirements due to limited resources and lack of regulatory expertise. Additionally, the focus in these environments is typically on scientific discovery and hypothesis testing, which can sometimes lead to overlooking practical aspects like scaling, quality control, and manufacturing capacity.

To ensure success, these groups need a comprehensive approach that balances the pursuit of scientific breakthroughs with practical considerations for development, such as creating a solid manufacturing process. Partnering with a reliable expert who can establish this process early on can help avoid scalability and regulatory challenges later. A well-designed manufacturing process, along with careful planning around regulatory strategies and cost management, significantly increases the likelihood of bringing a CGT product to market.

This whitepaper aims to highlight the avoidable costs—both in terms of time and budget—that arise from suboptimal decisions in early development. It will also address common pitfalls that early-stage scientists must be mindful of and key considerations for taking a CGT product from discovery to successful scale-up and regulatory approval. Finally, the whitepaper will examine the benefits of partnering with a specialized contract development and manufacturing organization (CDMO) early in the process to ensure the success of your program.