EP 175: Pioneering treatment for stop codon disease using tRNA with Michelle Werner of Alltrna

This week on The Genetics Podcast, Patrick is joined by Michelle Werner, CEO at Alltrna and CEO/Partner at Flagship Pioneering. They discuss Alltrna’s promising findings from its first preclinical study on using tRNA to rescue stop codon disease, the strategic use of basket trials, and more!Show Notes: 0:00 Intro to The Genetics Podcast01:00 Welcome to Michelle02:13 Overview of Alltrna’s aims and the advantages of using tRNA to tackle stop codon disease5:27 Using basket trials for genetic diseases08:03 Highlights from Alltrna’s first preclinical study using tRNA to restore protein production to clinically meaningful levels in methylmalonic acidemia (MMA) and phenylketonuria (PKU)14:02 Considerations in delivery techniques and Alltrna’s use of nanoparticles19:22 Stability of tRNA and how engineered tRNAs are recognized in vivo 23:12 Strategic design of basket trials and diseases that are covered26:16 Adaptive trial design in the rare genetic disease setting28:15 Michelle’s experience with regulatory organizations on new approaches to trial design32:14 Insights from spearheading Alltrna and Flagship Pioneering’s innovative approaches 37:26 Michelle’s lessons from working in big pharma versus a small biotech start-up40:50 Closing remarks and a call for collaboratorsFind out more Alltrna (https://www.alltrna.com/)Please consider rating and reviewing us on your chosen podcast listening platform! https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link