Challenges In Analytical Development And Testing For Gene Therapies

(Guest Column by Stephen Gacheru, Ph.D., Vice President, Analytical Development and Technical Operations at Center for Breakthrough Medicines)

The objective is confidence in our ability to develop and optimize assays specific to a specific investigational drug, used for a specific disease and delivered in a specific way.

The Center for Breakthrough Medicines approaches potency assays for gene therapies through a “matrix” approach that involves capturing two or more mechanisms of action to compile data with the goal of reaching a collective conclusion regarding final product potency. Our strategy involves the following assays:

• Virus product strength by ddPCR and infectivity through ddPCR. We have developed platform assays for these two techniques which we are now preparing for validation.
• Translation Potency via ddPCR. By following expression of the gene of interest to the mRNA stage, we can detect mRNA as part of early development potency assays.
• Expression of polypeptide chains via ELISA, using a representative green fluorescent protein sample analysis to set up a template assay design.
• Functional protein potency assay through ELISA, based on disease mechanism of action (MOA).

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