Alltrna, a Flagship Pioneering company, has received regulatory clearance to commence a Phase I clinical trial in Australia for its investigational transfer RNA (tRNA) therapy, AP003. This milestone marks the first time a tRNA therapeutic has entered human testing. The candidate is designed to treat liver diseases caused by a specific type of nonsense mutation, where a premature termination codon (PTC) leads to the production of a truncated, non-functional protein. AP003 is engineered to read through the PTC and insert the correct amino acid, aiming to restore the production of full-length, functional proteins.

The therapy utilises a liver-targeted lipid nanoparticle (LNP) delivery system. In preclinical models, AP003 demonstrated the ability to restore functional protein expression with a safety profile consistent with other oligonucleotide and LNP-based therapies. The Phase I trial will evaluate the safety and pharmacokinetics of a single ascending dose in healthy volunteers. The Arg-TGA mutation targeted by AP003 is the most common type of nonsense mutation in human genetic diseases.

PharmCube's NextBiopharm® database lists 15 other tRNA therapy candidates, all in preclinical stage. Click here to request a free trial for NextBiopharm®.