In glioblastoma, a deletion on chromosome 9 leads to the loss of the tumor suppressor gene CDKN2A. Smart RNA molecules alter their half-lives based on the CDKN2A expression, so that  production of the therapeutic effector protein is concentrated primarily in the tumor cells.  This selective response is enabled by the RNA’s engineered stability — it remains active and therapeutically effective only when CDKN2A is absent, and is rapidly degraded when the target is present, ensuring precise control and minimizing off-target effects.

In collaboration with our academic partners RXcel has now proven that this is no longer a hypothesis. We have successfully delivered RNA probes in vitro with conditional stability and delivered cytotoxic signals.