Session Chairs: Francois Gianelli, TreeFrog Therapeutics and Elaine Peters, Senior Leader in Cell and Gene Therapy Analytical Development

Testing for potency is expected for all biological medicinal products including ATMPs. Regulators expect potency assays will reflect the product’s mechanism of action (MOA) and, ideally, be predictive of the clinical response. The potency testing expectations for AAV and CAR-T products are relatively established. However, as more complex and novel modalities are being developed, there is still much to learn about what attributes are important for potency assurance.

In early phases of development, the mechanism of action of a new ATMP may not be fully understood. At early stage, developers face answering a number of questions: what should be measured for potency? What analytical methods should be used? How much characterisation data is needed? Starting multiple potency assay candidates’ development early in parallel to increasing knowledge of the MOA is the ideal approach. However, considering limited resources and test materials available early-on, it remains challenging to develop a fully relevant and yet efficient potency assay for early clinical trials.

This session will highlight pragmatic potency approaches for entering the clinic with novel therapeutic modalities while paving the way for later product development.

Session Speakers:

Case Study - Challenges and Opportunities in Developing Functional Dopamine Secretion Assays for Microtissues

Lucie Manache-Alberici, TreeFrog

Considerations for Potency Assay Development for Gene Editing Products

Ilya Shestopalov, Tessera Therapeutics

Additional Panelist:

Christina Grigoriadou, AstraZeneca

Elaine Peters

Appearances