Drug intelligence / Profile preview

Gln-TAG tRNA medicine

Development stage
Preclinical
Lead developer
Alltrna
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Gln-TAG tRNA medicine is an engineered transfer RNA (tRNA) therapeutic designed to treat genetic diseases caused by premature termination codons (PTCs), specifically the Glutamine-TAG (Gln-TAG) nonsense mutation. This innovative RNA therapy works by reading through premature stop codons in mRNA, allowing the translation machinery to continue producing full-length proteins despite the presence of the mutation. The engineered tRNA delivers the correct amino acid (glutamine) at the site of the TAG stop codon, thereby restoring production of functional proteins. This approach has shown promising results in both in vitro and in vivo studies, with machine learning optimization significantly improving the activity of these engineered tRNAs.

Other names
Glutamine to TAG tRNA medicineGlnUAG tRNA
02

Targets

PTC (50S ribosomal peptidyl transferase center)

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