Peter Emery-Billcliff
Principal Scientist Complement Tx
Seminars
Wednesday 6th May 2026
Developing a Potency Assurance Strategy for CTX001 from Early Clinical Entry to Late-Stage Readiness
9:45 am
- Outlining how early TCID₅₀ and mRNA assays were used to establish baseline infectivity and expression while planning for functional relevance as development progresses
- Developing an assay that assessed protein expression of the gene of interest, that better reflects biological effect
- Discussing planned assay modifications to enable increased robustness, lifecycle management, and eventual transition towards a bioactivity-focused potency assay for later-phase development
