November 30, 2022

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Flagship bet on tRNA therapy which can treat thousands of genetic diseases

Flagship bet on tRNA therapy which can treat thousands of genetic diseases



Flagship bet on tRNA therapy which can treat thousands of genetic diseases.

After Moderna’s great success, Flagship bet on tRNA therapy, which can treat thousands of genetic diseases.

According to the central principle , DNA is transcribed into mRNA and mRNA is translated into protein . This is the flow direction of genetic information. Gene therapy for DNA, vaccines for mRNA, and targeted degradation of proteins have received a lot of attention in recent years. Many breakthroughs have been made.

However, tRNA (transfer RNA) , which is very important in the flow of genetic information, seems to have been overlooked. tRNA is the key performer in the translation of mRNA into protein. The amino acid corresponding to this codon is transferred to the polypeptide chain during ribosome synthesis, thereby realizing the translation process from mRNA to protein.

Flagship bet on tRNA therapy which can treat thousands of genetic diseases

In 2010, the famous biotech venture capital firm Flagship Pioneering CEO of the Nuba · A Feillant (Noubar Afeyan) sees great potential therapy mRNA, and Derek Rossi (Derrick Rossi) and Robert Langer (Robert Langer) co-founded Moderna . The success of the mRNA COVID-19 vaccine has made mRNA technology one of the most important technological breakthroughs in this century.

Now, Flagship has set its sights on tRNA and invested $50 million to establish a new startup company: Alltrna .

Dr. Lovisa Afzelius , CEO of Alltrna , said that tRNA drugs have the potential to treat thousands of diseases . TRNA drugs have not yet been tested in humans. Lovisa Afzelius has remained silent on which diseases Alltrna will conduct research first, but said that it will target a series of rare and common genetic diseases.

tRNA is a molecule that has evolved and has been conserved for billions of years. It is the key executor of mRNA translation into protein. Without tRNA, protein cannot be made, but tRNA has never attracted enough attention.

tRNA can also produce various modifications, which have therapeutic prospects for genetic diseases. If tRNA is used as a programmable drug, it can theoretically control anything related to protein translation.

More than 11% of genetic diseases are caused by gene mutations leading to early stop codons and early termination of protein translation, such as cystic fibrosis . In response to this, design an inhibitory tRNA that carries the correct amino acid and inserts the correct amino acid at the stop codon in advance to enable the protein to continue to be translated, thereby producing a protein with normal function.

Flagship bet on tRNA therapy which can treat thousands of genetic diseases

Prior to this, ReCode Therapeutics , established in 2015, has been exploring the use of tRNA for the treatment of cystic fibrosis.

Reference:
https://www.fiercebiotech.com/biotech/flagship-unveils-its-next-rna-bet-50m-for-alltrna-to-explore-thousands-genetic-diseases

Flagship bet on tRNA therapy which can treat thousands of genetic diseases

(source:internet, reference only)


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