Module 9 2024

03/09/2024

Logistics: Co-Ordination Between Patient Care, Source Material Collection Site, Manufacturing and Testing

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Potency

Monogenic inherited diseases: transgene expression provides indication of defect correction Oncology and CAR+T are a special, difficult case for potency ● Elusive MoA ● Role of TME and suppressive mechanisms CAR-T (CAR expression is not a potency assay!!!) ● In-vitro tumour killing assay ● Cytokine (IFN-G) expression following CAR-T stimulation ● Difficult valuation, long TAT and poor association with clinical outcome New technologies (omics) may help to deep dive into cell biology and find better potency marker

Incremental implementation Orthogonal approach: no single test can measure attribute predicting efficacy In place prior starting pivotal trials, used comparability and stability studies Validation in place prior BLA A wide range of product attribute measured for characterization not necessarily become a spec for release Potency assurance as comprehensive strategy as a multifaceted approach include process design and control, material control, in-process testing and potency lot release assay Potency assay strategy described in IND.

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