EP 139: Rare disease breakthroughs using tRNA with Michelle Werner, CEO of Alltrna and CEO-Partner of Flagship Pioneering
June 20, 2024
This week, we welcome Michelle Werner, CEO of Alltrna and CEO/Partner at Flagship Pioneering. Michelle and her team are working at the cutting-edge of tRNA therapies, which they hope will present a scalable treatment method for people in the rare and ultra-rare disease communities.
Join Patrick and Michelle for an exciting conversation about this breakthrough technology, from the transferable potential of this new modality across stop codon diseases, to Michelle’s personal passion for delivering workable solutions to the rare disease community.
0:00 Intro to The Genetics Podcast.
01:00 Welcome to Michelle.
02:00 Sstop codon diseases and how are they characterised
03:45 Diseases caused by premature stop codons in haploinsufficient genes.
04:35 The role of transfer RNA technology in finding solutions for premature stop codon diseases.
06:16 How Alltrna is engineering tRNAs which can bind to premature termination codons so healthy coding can be restored.
08:10 The number of different engineered tRNAs required to address the 6,000+ genetic diseases
09:38 How Alltrna aims to deliver precision repair using just 19 amino acids.
12:10 Lipid nanoparticles: Modes of delivery and how to get the technology to the right tissues.
14:20 Major types of delivery, from Adeno-Associated Virus (AVV) to conjugations.
16:30 The development of tRNA as a new therapeutic approach and the latest foundational tools.
18:38 How basket clinical trials in the oncology space are enabling mutation specific therapies.
20:22 Entering into the rare genetic liver disease space with a basket trial approach.
21:40 tRNA opening up a unique opportunity to target multiple rare and ultra-rare diseases.
23:13 Measuring efficacy when using an umbrella approach to stop codon diseases.
26:25 Tackling conversations with the Food and Drug Administration and other health authorities.
29:46 How proving the modality to be safe and effective opens up the possibility to focus on diseases which are less well characterised.
31:18 The importance of patient advocacy groups and industry collaboration for delivering therapies and technologies across different geographies.
32:40 Decision-making, including when to develop inhouse and when to seek external partnerships.
34:42 Key learnings Michelle has brought from large pharma organisations to Alltrna
37:51 Working with patient-led organisations to power development in disease areas where there is less commercial value.
40:21 Why Michelle is so passionate about the work being done at Alltrna and how the tRNA modality can bring new solutions to rare disease families and patients.
41:28 One technology or idea Michelle thinks everyone should keep watching and learn more about.
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