A small, highly virtualized biotech had identified an attractive target in fibrotic diseases and needed a translational strategy to generate clinical data that enables data-driven decision-making for future development as well as increases the asset value.
TherapeutAix was contracted to define the translational approach scoping elements ranging from in vitro / in vivo mechanistic bridging through to disease models with translational integrity.
We have reviewed the literature and revisited earlier internal decision matrixes enabling prioritization between different fibrotic diseases. A broad translational plan was outlined, comprising several distinct and partly overlapping trajectories. Several sets of in vitro, in vivo, and clinical translational study concepts were created that emphasized different aspects of translational confidence (e.g., mechanistic vs antifibrotic/phenotypic).
This translational concept was discussed with the client, and a confidence-generating and value-optimized set of studies were chosen that are now further refined.