ONE GENETIC MEDICINE.
THOUSANDS OF DISEASES.

Arginine to TGA (Arg-TGA) liver stop codon disease can affect patients with Arg-TGA mutations in any genetic disease of the liver, including phenylketonuria, organic acidemias, and urea cycle disorders. The Arg-TGA mutation is the most prevalent nonsense mutation in the liver.

AP003 is a chemically modified, engineered tRNA oligonucleotide encapsulated in a clinically validated, liver-directed lipid nanoparticle (LNP) for the treatment of patients with liver Stop Codon Disease that carry an Arg-TGA mutation.
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AP003 is in clinical evaluation in healthy volunteers in Australia. This study is an important step toward a potential Phase 2 basket trial that aims to include patients across multiple liver inborn errors of metabolism driven by Arg-TGA variants, including PKU, organic acidemias, and urea cycle disorders.

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Glutamine to TAG (Gln-TAG) liver stop codon disease can affect patients with Gln-TAG mutations in any genetic disease of the liver, including phenylketonuria, organic acidemias, and urea cycle disorders.

Alltrna’s engineered tRNA are designed to precisely replace the correct amino acid that would be present without a nonsense mutation. Future programs are intended to bring forward tRNA medicines to address other stop codon liver disease patients based on their nonsense mutation type.