Precision-RNA is pioneering an innovative platform to bring personalized RNA-based treatments into clinics - starting with severe, currently untreatable brain diseases. Using programmable molecules called antisense oligonucleotides (ASOs), the platform can be tailored to each patient’s unique genetic mutation, yet remains scalable by applying a shared biological mechanism across many rare diseases. This innovative approach has already led to two individualized treatments for the rare neurological disease Ataxia-Telangiectasia, now in clinical use. By combining precision and scalability, PRECISION-RNA aims to revolutionize drug development for rare conditions - and pave the way for future individualized therapies in common diseases as well.

Further information here.