Early-phase operational readiness gaps in oncology cell and gene therapy trials and implications for scalable trial infrastructure.

Abstract

11067 Background: First-in-human (FIH) and Phase 1 oncology cell and gene therapy (CGT) trials have highlighted that operational readiness, rather than scientific feasibility, is often the primary constraint on trial execution and patient access. Many clinical sites lack the infrastructure, staffing, and process maturity required to support CGT trials, limiting enrollment and scalability. Methods: Operational lessons were analyzed across multiple early-phase oncology CGT programs, including autologous and allogeneic platforms. Assessments focused on trial execution during FIH and Phase 1 activation, site readiness, vector and cellular product handling, storage and chain-of-custody requirements, coordination among manufacturing, sites, and couriers, staffing and training needs, and turnaround times from leukapheresis or biopsy to dosing. Results: Consistent site readiness gaps were identified in apheresis coordination, vector storage and handling, chain-of-custody documentation, and real-time cross-functional communication. Autologous programs showed increased operational complexity driven by patient-specific manufacturing, narrow scheduling windows, and variable vein-to-vein turnaround times. Allogeneic programs had reduced scheduling constraints but introduced challenges related to centralized manufacturing oversight and site-level accountability. Across platforms, CGT trials required specialized staffing and training beyond standard oncology research models. Insufficient early operational enablement resulted in delayed dosing and reduced patient access by ~1-3 months. Conclusions: Lessons from early-phase oncology CGT trials demonstrate that proactive operational readiness, particularly at the site level, is essential to enable scalable trial infrastructure and equitable patient access. Early readiness assessments, targeted workforce training, and integrated manufacturing-logistics coordination are critical to support efficient CGT trial execution and future expansion. Learning Objectives: Recognize common operational readiness gaps identified during FIH and Phase 1 oncology CGT trials. Understand how manufacturing, logistics, and site coordination impact early-phase trial execution. Apply early operational learnings to inform scalable CGT trial infrastructure. Key Takeaways: Operational readiness is a primary determinant of early CGT trial success. Many sites require significant enablement to conduct CGT trials effectively. Early investment in readiness improves turnaround times and patient access.