Suppressor tRNAs: Giving genetic medicines a broader reach
From preclinical therapies to David Liu’s one-time approach, new tools aim to bypass stop codons across a wide range of diseases
While 2025 saw one tRNA biotech shut its doors, at least three others are still betting that engineered suppressor tRNAs can rescue protein production in patients with stop codon mutations.
The field has received a new boost from advances in other genetic disease modalities, novel research from academic labs solving some longstanding problems, and an injection of star power from base and prime editing pioneer David Liu who is turning his technology toward a one-and-done approach that he labels “gene-agnostic base editing.” (Liu discussed this strategy, and others, with BioCentury in an October Q&A). ...