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Beyond Biotech podcast 66 – treating rare diseases with tRNA

Beyond Biotech podcast 66 – treating rare diseases with tRNA

FromBeyond Biotech - the podcast from Labiotech


Beyond Biotech podcast 66 – treating rare diseases with tRNA

FromBeyond Biotech - the podcast from Labiotech

ratings:
Length:
32 minutes
Released:
Oct 6, 2023
Format:
Podcast episode

Description

Compared to the smallest mRNA molecule, which is 300 nucleotides long, the largest transfer RNAs (tRNA) is less than a third of the size at 76 nucleotides. Scientists at Alltrna are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic mutation. This year, Alltrna presented at ASGCT the first demonstration that an engineered, modified tRNA could recognize and correct, in vivo, a flawed mRNA instruction no matter where it occurred in the genome.Alltrna, which was founded in 2018 by Flagship Pioneering, recently announced it had raised $109 million in a Series B financing to advance the company’s platform and first drug candidates towards the clinic for a first indication in Stop Codon Disease.  Stop Codon Disease encompasses ​​thousands of rare and common diseases that arise from premature termination codons (PTC), also known as nonsense mutations, where the code for an amino acid has been mutated into a premature “stop” codon. This results in a truncated or shortened protein product with no or altered biological activity that causes disease. Approximately 10% of all people with a genetic disease have Stop Codon Disease, representing approximately 30 million people worldwide. Alltrna’s tRNA medicines can read these PTC mutations and deliver the desired amino acid, thereby restoring the production of the full-length protein.The company’s platform incorporates artificial intelligence and machine learning tools to ‘learn’ the tRNA language and deliver diverse, programmable molecules with broad therapeutic potential. This week, our conversation is with Alltrna CEO, Michelle Werner.
Released:
Oct 6, 2023
Format:
Podcast episode

Titles in the series (95)

Welcome to the official Labiotech.eu podcast - Beyond Biotech! Each week, we talk about what's happening in the world of biotech, with news and interviews with experts from companies around the world. Join us as we cover the latest news, breakthroughs and innovations shaping the life sciences industry.A new podcast episode is available every Friday.The host is Jim Cornall.