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

The Genetics Podcast

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 Podcast

01:00 Welcome to Michelle

02:13 Overview of Alltrna’s aims and the advantages of using tRNA to tackle stop codon disease

5:27 Using basket trials for genetic diseases

08:03 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 nanoparticles

19:22 Stability of tRNA and how engineered tRNAs are recognized in vivo

23:12 Strategic design of basket trials and diseases that are covered

26:16 Adaptive trial design in the rare genetic disease setting

28:15 Michelle’s experience with regulatory organizations on new approaches to trial design

32:14 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-up

40:50 Closing remarks and a call for collaborators

Find out more

• Alltrna (https://www.alltrna.com/)

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