CERNAIS Presents Innovative RNA-Based Therapeutic Strategies at the 2025 LouLou Foundation Meeting
The CERNAIS team was honored to present its latest research at the 2025 LouLou Foundation Meeting, held on October 27–28 at the Royal Sonesta Hotel. This event brings together leading scientists, clinicians, and organizations dedicated to advancing research and finding treatments for CDKL5 Deficiency Disorder (CDD) and related neurodevelopmental diseases.
Our team, presented a poster entitled “New targeted therapies for the treatment of rare diseases,” showcasing the latest progress in CERNAIS’s RNA-modifying molecule (RMM) platform. The poster summarized our ongoing efforts to identify and optimize small molecules that can reactivate silenced genes on the inactive X chromosome — a promising therapeutic avenue for CDD and other X-linked genetic conditions. At CERNAIS, we combine artificial intelligence, RNA structural modeling, and cellular assays to discover small molecules that act directly on RNA. This approach allows us to target diseases previously considered undruggable, bridging computational predictions with experimental validation to accelerate the development of RNA-based therapeutics.
The LouLou Foundation Meeting provided an inspiring environment to share our advances and exchange ideas with the broader CDKL5 research community. The discussions reinforced our belief that cross-disciplinary collaboration between academia, biotech, and patient-driven organizations is key to translating innovative science into effective therapies.
We extend our gratitude to the LouLou Foundation for fostering such a dynamic and collaborative ecosystem. Their commitment to supporting research and innovation continues to inspire our mission to develop first-in-class, RNA-targeted small molecules for rare neurodevelopmental disorders.
Stay tuned for more updates as CERNAIS continues to expand its pipeline and partnerships toward the next generation of RNA-based medicines.
