EP 139: Rare disease breakthroughs using tRNA with Michelle Werner, CEO of Alltrna and CEO-Partner of Flagship Pioneering
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0:00 Intro to The Genetics Podcast.01:00 Welcome to Michelle.02:00 Sstop codon diseases and how are they characterised03:45 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 diseases09:38 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 Alltrna37:51 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.Please consider rating and reviewing) us on your chosen podcast listening platform!Find out more:alltrna.com)rarediseaserenegades.org)