Module 9 2021

Structure ● VCN should be justified in relation to safety and intended use ● Off-target modification and integration analysis ● Vector and cell identity by sequenceing and indel characterization ● Non cellular components ● Potency assay to be developed fron FIH Impurities ● When a specific cell type is required or selelcted, cell types for therpeutic be defined and amount controlled ● Fold reduction assessment may avoid routine testeing for selected process simpurities ● Persistence of gene editing tools ● Adventitious and endogenous viruses including RCV for GTMPs Quality Control and Assay validation ● Two step relase program when release data avalible only after administration ● Reference batch to be assigned whenever possibile ● Limited (safety) assay validation for FIH, potency assay in place and validated for MAA ● Potency assay: quantitative measurement of newly acquired characteristics Characterization and Control

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