CERNAIS Showcases Innovative RNA-Based Therapeutics at MoML 2025 @ MIT
The CERNAIS team is delighted to share that our team, presented our latest data at the Mechanisms of Molecular Logic (MoML) 2025 meeting, held at the Massachusetts Institute of Technology (MIT).
The poster, titled “New targeted therapies for the treatment X-linked genetic disorders,” highlighted the progress of our proprietary RNA-modifying molecule (RMM) platform, which combines artificial intelligence, RNA structure prediction, and experimental validation to identify small molecules capable of reactivating silenced genes. This work represents a concrete step toward new therapeutic options for rare X-linked neurodevelopmental diseases such as Rett Syndrome and CDKL5 Deficiency Disorder.
At CERNAIS, we are building a bridge between computation and biology. Our AI-powered pipelines analyze RNA structures, predict RNA–small molecule interactions, and guide the discovery of novel therapeutics that act on RNA rather than DNA or proteins. This approach allows us to target diseases that have remained beyond the reach of traditional drug discovery.
Presenting our findings at MIT was an inspiring opportunity to engage with an international community of scientists working at the intersection of molecular biology, chemistry, and artificial intelligence. The discussions at MoML 2025 reinforced the importance of RNA- targeted therapies as one of the most promising frontiers in biomedicine. Our participation in this event reflects CERNAIS’s growing commitment to combining academic excellence with translational innovation. We are proud of our multidisciplinary team — spanning computational modeling, cell biology, and medicinal chemistry — and of our ongoing collaborations with academic and clinical partners who share our mission: to design small molecules that can correct gene expression at its root.
We thank Prof. Barzilay, the MoML organizers and attendees for the stimulating discussions and valuable feedback. This experience will help us refine our strategies and accelerate our journey toward the development of first-in-class RNA-modifying therapeutics for rare genetic disorders. Stay tuned for our upcoming updates as CERNAIS continues to expand its research and preclinical pipeline in RNA-based precision medicine.
