Presentation (CTI)

OBJECTIVES: This study examines the business case for point-of-care (POC) production of personalized gene therapy (GT) with and without “Nanospresso” (NP), a closed-system platform technology for frequently administered mRNA-based therapeutics. They pose significant operational challenges that limit access and likely keep prices high—especially for rare and orphan diseases. Platform-based approaches enabling POC utilising personalised APIs could standardize production across multiple rare diseases, and therefore offer tailored patient access to otherwise unavailable treatments.
METHODS: We used Time-Driven Activity-Based Costing (TDABC) and structured expert elicitation (SEE) to develop and assess a standardized mRNA-based gene therapy production process, following good medical practice (GMP) guidelines. The standardized process was informed by interviews with pharmacists with compounding expertise and then quantified using time estimates. Workload and costs were calculated across scenarios (e.g., with/without NP, with/without in-house mRNA synthesis), and integrated into an interactive tool that models alternative scenarios.
RESULTS: Preliminary results suggest manual POC production requires an average of 41 hours of pharmacist or qualified person time per dose compounded, with a cost of €4030 (excluding the purchased API like mRNA). For bi-weekly delivery (e.g., metabolic liver diseases), this results in 7634 hours of annual work and equipment usage (€96k)—79 hours per month, per patient. NP-supported production reduces labour by 22 hours, and reduces equipment requirements, lowering costs to €1020. This cuts workload to 3619 (37) hours annually (monthly). Other modelled scenarios include in-house or centralized mRNA synthesis, significantly reducing overall cost due less quality assurance requirements before medication release.
CONCLUSIONS: Closed-system technologies like NP could automate elements of CGT compounding, reducing operational strain and workload. In this scenario, hospitals may deliver therapies more efficiently, easing workload, and reducing the liklihood of errors. To enable adoption and patient access, infrastructure investments are essential as mRNA-based GTs move beyond clinical trials.