A | FOR IT, IT SEEMS THAT FOR MANY OF THE PLATFORMS NEEDED to manage a sequence-based modality such as gene therapy, one would have use cases similar to current modalities (e.g., small molecules) — storage, tracking, bar coding, assay input/output, data capture, and the like. For informatics, gene therapy will be different from other platforms. For example, with small molecules, you may use informatics for target specificity, QSAR (quantitative structure–activity relationship) models, and docking. But for gene therapy, you would use informatics for understanding vector safety, integration analysis, immunogenicity, gene expression, and off-target effects. I am aware of only a small number of institutions/companies that have built leading informatics capabilities for this exciting new frontier of sequence-based modalities.
JOHN REYNDERS is VP, R&D strategy, program management, and data sciences at Alexion.