Transfer RNA (tRNA) developer Alltrna announced on Tuesday that it has received approval to initiate a Phase 1 clinical trial of AP003 in healthy volunteers in Australia.

The study has been approved under Australia's Therapeutic Goods Administration (TGA) Clinical Trial Notification (CTN) scheme, following review by the Human Research Ethics Committee (HREC).

AP003 is a chemically modified, engineered tRNA therapeutic designed to restore full length protein production by inserting the correct amino acid at an Arg-TGA premature termination codon (PTC). It is claimed to be the first tRNA therapeutic to advance into clinical trials, marking a foundational milestone for the field and for patients with Stop Codon Disease.

Stop Codon Disease encompasses thousands of rare and common diseases that stem from PTCs, also called nonsense mutations, where the code for an amino acid has been mutated into a premature "stop" codon. This results in a truncated or shortened protein product with no or altered biological activity that causes disease.

Alltrna says that it in preclinical models, AP003 has demonstrated the ability to restore full-length protein expression and downstream functional activity in disease settings driven by shared PTCs, supporting the biological validity of a mechanism-based approach across genes. Preclinical studies to date show a safety profile that is consistent with established experience for oligonucleotide and lipid nanoparticle-based therapies, supporting clinical evaluation. Together, these data support Alltrna's broader clinical strategy to evaluate AP003 across multiple diseases driven by shared PTCs.

The Phase 1 study is designed to evaluate the safety and pharmacokinetics of single ascending doses of AP003 in healthy volunteers. Data from this study are expected to inform subsequent clinical development, including studies in genetically defined patient populations.