Using basket trials for tRNA therapies

Synopsis: At JPM 2026 in San Francisco, Alok Tayi welcomes Michelle Werner, CEO of Alltrna, to Biotech 2050 for a powerful conversation at the intersection of personal mission, platform biology, and rare-disease drug development. Michelle traces her two-decade career across Bristol Myers Squibb, AstraZeneca, and Novartis—and the moment everything changed when her child was diagnosed with a rare disease. That experience led her to Alltrna and its pioneering engineered tRNA platform, designed to correct nonsense mutations across hundreds—potentially thousands—of genetic disorders with a single therapeutic approach. Together, Alok and Michelle explore how tRNAs work, why “stop-codon disease” could redefine rare-disease classification, and how basket trials borrowed from oncology may accelerate development. They dive into delivery strategy, portfolio expansion into CNS and muscle disorders, regulatory innovation, and how AI is reshaping molecular design—offering a rare look at what it takes to build a first-in-class modality from the ground up. Biography: Michelle is a seasoned pharmaceutical executive with more than 20 years in the industry spanning commercial and research & development (R&D) responsibilities. Prior to Alltrna, Michelle served as Worldwide Franchise Head, Solid Tumors at Novartis Oncology, where she was responsible for delivering the disease area strategies across multiple tumors and led business development efforts resulting in a doubling of long-term portfolio value for the franchise. Previous to Novartis, Michelle was a senior leader at AstraZeneca and as Global Franchise Head in Hematology, she was critical in launching multiple indications worldwide for CALQUENCE®. Prior to this, Michelle was Head of US Oncology, where she led the business through dramatic growth in both team and revenue through eight-plus product launches. Previous to AstraZeneca, Michelle was with Bristol-Myers Squibb for 10 years in various positions of increasing responsibility including roles in sales, marketing, and market access in the US and UK, and above market in Europe (based in France) and global almost exclusively in oncology. Michelle started her professional career in R&D, working hands-on with patients at the Oncology Clinical Trials Unit at Harvard Medical School before moving into industry in clinical operations. Outside of her corporate responsibilities, Michelle is a wife and mother to three children and is a member of the rare disease community. She is currently serving a Board appointment for the non-profit organization Rare Disease Renegades, a purpose that fuels her passions both personally and professionally.