AP003

AP003 is a chemically modified, engineered transfer RNA (tRNA) oligonucleotide developed by Alltrna for the treatment of genetic diseases caused by premature termination codons (PTCs), specifically the arginine to TGA (Arg-TGA) nonsense mutation. This mutation occurs when a codon encoding arginine mutates to TGA, resulting in early termination of protein synthesis and loss of functional protein. AP003 is formulated in a clinically validated, liver-directed lipid nanoparticle (LNP) delivery system. Its mechanism involves readthrough of the Arg-TGA stop codon during translation, enabling insertion of arginine at the site and restoration of full-length protein production. Preclinical studies have demonstrated that a single dose restores clinically meaningful levels of target proteins in mouse models for methylmalonic acidemia (MMA) and phenylketonuria (PKU), two rare metabolic disorders driven by this specific PTC[1][2][4][5]. The approach supports development across multiple rare genetic liver diseases sharing this mutation.

Other names

Arg-TGA tRNA medicineengineered tRNA for Arg-TGA PTCAlltrna Arg-TGA tRNA candidate