Many severe diseases still lack effective treatment. Where therapies exist, they often hit healthy cells too – and side effects limit the dose patients can receive. An seRNA construct is activated at the RNA level: it is delivered inactive and switches on only when it detects a disease-specific RNA inside the target cell. In healthy cells, it stays silent. This allows more potent therapies against diseases with high unmet medical need, while sparing healthy tissue.
Because sensor and effector are interchangeable, the same platform can eliminate a diseased cell – or restore its function.