Top Considerations for CGT Development: Takeaways from Phacilitate Advanced Therapies Week 2023

As science and technology swiftly advance, the development of cell and gene therapies (CGT) has accelerated. With this growth comes a myriad of considerations and challenges, which were discussed at the 2023 Phacilitate Advanced Therapies Week. Syneos Health attended the event to track industry developments and harvest insights that can help shape the future of cell and gene therapy (CGT) drug development for better patient outcomes. Here are some of the key takeaways from the event:  

1. As projected, more adoptive cell therapies are gaining regulatory approval, however, the industry is not confident that healthcare systems are ready to deliver these treatments in a commercial setting 

Due to the complex delivery requirements for commercially available adoptive cell therapies, we are currently unable to treat patient populations. This challenge is expected to become more acute, as more therapies become commercially available in a broader range of indications, and treatments are used in earlier lines of treatment (e.g. second-line therapy for large B-cell lymphoma). Compounding this issue, healthcare system stakeholders don’t feel like their voices are being heard, making it harder to coordinate the improvements required in the healthcare system.

2. The race is on to get in-vivo gene modified cellular immunotherapy into clinics  

With the promising data emerging in pre-clinical studies, developers are keen to understand if the treatments will translate to humans—creating a burgeoning field of companies developing therapies at the pre-clinical stage. Excitement is high due to the possibility of removing logistical complications while maintaining clinical efficacy, such as avoiding transportation and ex-vivo manufacturing of patient's cells.  

3. While the industry understands that product development needs to be done differently for CGT—including key activities being conducted much earlier in development—it’s unclear whether the funding landscape can facilitate this level of rigor 

To receive reimbursement and gain uptake from patients, endpoints must be designed beyond achieving regulatory approval, demonstrating how these treatments change a patient’s quality of life. For a product to be commercially viable, a manufacturing strategy that minimizes the cost of goods must be implemented. The investment community is rightly focused on the quality of science, but to enable winners it must promote CGT focused portfolio / product development planning.  

4. If it feels like there are conflicting messages about progress and prospects for CGT, it’s because it is often used as a catch-all term for a diverse group of emerging technologies 

2022 was another breakthrough year in the approval of CGTs. However, some larger companies appear to be showing signs of caution. Complicating perceptions are the multitude of technologies being developed that can be considered a CGT (e.g., gene therapy, gene editing, RNA / DNA targeting therapies, etc.). If you map these therapy types on the Gartner Hype Cycle (a graphical representation of the maturity, adoption, and social application of specific technologies) there would be a significant spread, explaining the mixed messaging in the industry.  

5. There is limited consensus and a fragmented approach to how these technologies should be communicated with the public to promote adoption and uptake 

Awareness of challenges with public adoption and perception of CGT are front-of-mind after the roll-out of mRNA vaccines to treat COVID-19. Patient advocacy groups are playing a critical role in providing treatment information; however, this can come with its own challenges for sponsors as they determine how to disseminate information. Reporting in mainstream media is rapidly increasing, yet there is no consensus on whether and how to control messaging going forward.  

We understand that these clinical trials often involve treatment aspects that are novel to investigators, site staff and patients alike, involve very complex logistical considerations and are taking place in a rapidly evolving regulatory landscape. To learn more or partner with us in the development of your CGT product, contact the Syneos Health Cell and Gene Therapy Development Engine today. 

Contributors

Pete Robinson, VP, Medical & Scientific Strategy & Head of Cell and Gene Therapy, Syneos Health

James Barwick-Silk, Director, Medical & Scientific Strategy, Syneos Health

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