Podcast: Treating Rare Diseases With tRNA

In this episode of the In Vivo podcast Michelle Werner, CEO of Alltrna, discusses using tRNA as a therapeutic modality for rare diseases.

In Vivo Podcast

Michelle Werner left a long career in Big Pharma to lead Alltrna, a Flagship Pioneering company focused on treating rare diseases with tRNA therapeutics.

Michelle Werner
Michelle Werner • Source: Michelle Werner

Werner spent 20 years as a pharma exec, she most recently served as worldwide franchise head for Novartis Oncology’s solid tumors division, where she doubled the franchise’s portfolio value. At AstraZeneca, she launched multiple indications for Calquence (acalabrutinib) and led US Oncology through significant growth. She also held senior roles at Bristol Myers Squibb and began her career in R&D at Harvard Medical School.

Outside of work, she is a wife, mother of three, and active in the rare disease community, serving on the Board of Rare Disease Renegades.

In this episode of the In Vivo podcast, Werner divulges the very personal impetus for the switch to biotech, explains why tRNA as a therapeutic modality has been overlooked until now, and the promise it holds for treating diseases stemming from nonsense mutations.

She discusses Alltrna’s development strategy, preclinical findings to date, the company’s finances, venture capital interest in tRNA, and differences between working in Big Pharma and a biotech.

Timestamps:

1:00 - Michelle’s background in big pharma and her decision to switch to a biotech focused on rare diseases

4:15 - The origins of Alltrna

6:15 - The Flagship Pioneering company origination model

7:15 - The function and promise of tRNA and why it has been overlooked to date

10:40 - The Alltrna platform, machine learning, and nonsense mutations

14:10 - Alltrna’s development strategy: prioritizing liver diseases for now

17:30 - Promising preclinical findings show protein rescue

21:10 - The advantages of using a basket trial study design in rare diseases

25:55 - Beyond nonsense mutations

27:00 - Company finances and investor interest in tRNA therapeutics

28:35 - The merits of working in biotech vs. pharma

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