Nonsense! Engineered tRNAs Offer Universal Solutions to Stop Codon Diseases

In May of 2020, drug developer Michelle Werner, PhD, learned that one of her three children—her middle child, Caffrey—was diagnosed with Duchenne muscular dystrophy (DMD). Werner went searching for an existing or experimental cure but came out empty-handed. So, after a couple of decades as a big pharma stalwart, Werner made a major career change and took over as CEO of Flagship Pioneering company Alltrna, which had a solution she believed could help children and families in similar positions—even if not Caffrey himself.

In this Behind the Breakthroughs episode, Werner talks about the experience of being a mother to a child with a rare disease and her mission as Alltrna's CEO to find cures for all rare diseases, the majority of which have no approved therapies. Michelle goes in-depth into the mechanism behind Alltrna’s engineered transfer RNAs (tRNAs) and their broad applicability to all diseases caused by nonsense mutations leading to premature stop codons.

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Produced by Bill Levine

Hosted by Jonathan D. Grinstein

Audio mixed and mastered by Jason Mack

Music provided by Artem Hramushkin

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