Ep. 254, Chapter 3: What's A Stop Codon Disease? With Michelle Werner

30:03-42:15

Welch and Werner discuss Alltrna's patient-first mindset and its practical implications for early-stage rare disease companies. Werner goes on to define the concept of a "stop codon disease" as a reclassification for patients with genetic diseases stemming from a premature termination codon (nonsense mutation). This reclassification, affecting approximately 10% of genetic diagnoses, could allow a single engineered tRNA therapy to address thousands of different life-threatening conditions, offering a universal solution instead of a disease-by-disease approach. Alltrna leverages AI and machine learning to navigate the vast combinatorial space of tRNA designs, optimizing their sequence and chemical modifications for enhanced activity and stability.