February 29th 2024 marks not only the rarest day of the year but also the Rare Disease Day, dedicated to raising awareness and generating change for the 300 million people worldwide living with a rare disease, along with their families and carers. With over 7,000 rare diseases identified, achieving equity for those affected means ensuring access to diagnosis, treatment, health, social care and opportunity.
NovAliX and Urania Therapeutics join forces to develop new, safe, and effective readthrough compounds that hold promise for treating rare diseases.
Urania Therapeutics specializes in protein repair, targeting genetic diseases caused by nonsense mutations leading to premature termination of protein synthesis. Their approach involves structure-based drug design to create efficient and safe “readthrough” compounds, restoring the synthesis of the missing full-length protein and its physiological functions. As readthrough is a gene-independent mechanism, Urania Therapeutics’ molecules have potential applications across multiple rare disease indications.
NovAliX engages in drug discovery alliances with biotechnology companies. We make available our expertise in medicinal chemistry, biology and biophysical technologies through risk-sharing or in-kind contributions.
Share your colours with us! Get also involved in #RareDiseaseDay.
