Wrapping up 2023: A recap of top biotech moments of this year
Another area of significant biotech breakthrough in 2023 is the development of new weight-loss drugs called GLP-1 analogues – which mimic the ...
Companies like Alltrna
Another interesting development is the use of RNA technology in treating rare diseases. <anchor>Companies like Alltrna</anchor> are exploring the potential of transfer RNAs (tRNA) to treat a wide range of rare diseases. This approach leverages the unique biology of tRNAs and represents a novel direction in the field of RNA therapeutics​​.
Beyond Biotech podcast 66 – treating rare diseases with tRNA
Beyond Biotech podcast 66 – treating rare diseases with tRNA

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[Home](https://www.labiotech.eu/)[Podcast](https://www.labiotech.eu/podcast/)**Beyond Biotech podcast 66 – treating rare diseases with tRNA**

# Beyond Biotech podcast 66 – treating rare diseases with tRNA

![Jim Cornall](data:image/svg+xml,%3Csvg%20xmlns=%22http://www.w3.org/2000/svg%22%20viewBox=%220%200%20103%20103%22%3E%3C/svg%3E)

By [Jim Cornall](https://www.labiotech.eu/author/jimcornall/)

2 minutesmins

October 6, 2023-Updated:  onMay 23, 2024

2 minutesmins

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**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.

Explore other topics:[Rare disease](https://www.labiotech.eu/tag/rare-disease/)[RNA technology](https://www.labiotech.eu/tag/rna-technology/)[USA](https://www.labiotech.eu/tag/usa/)

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