Alltrna Announces First-in-Class tRNA Therapy AP003 Enters Clinical Trials
Alltrna
tRNA Therapy Developer
3Month31Day, byFlagship PioneeringFoundedtRNATherapy Leader CompanyAlltrnaAnnounced that it has been approved for launch in Australia AP003 TheIPhase clinical trial. It is claimed that this is the first to enter the clinical trial stage. tRNA Therapy, marking an important milestone in this field.
AsmRNAAn auxiliary molecule in the translation process,tRNACarrying specific amino acids, through the anticodon withmRNAThe codon on the chain binds, transferring the corresponding amino acid to the ribosome to complete the elongation of the polypeptide chain. Nonsense mutation (nonsense mutation), i.e., a codon for a particular amino acid has mutated into a premature stop codon (premature termination codons，PTC), leading to premature termination of peptide chain synthesis and incomplete corresponding protein product.(Truncate or Shorten)And dysfunction(Loss or alteration of biological activity), thereby leading to termination codon diseases (Stop Codon Disease）。
Termination codon diseases encompass thousands of rare and common diseases.Alltrna Developing the ability to identify PTCOf tRNA The drug can deliver the required amino acids to cells, thereby restoring the production of full-length proteins.
AP003 Is a chemically modified and engineeredtRNAOligonucleotides, delivery system targets the liverLNP。AP003Being developed to treat liver stop codon diseases, which are caused by the "arginine to stop codon" mutation (arginine-to-TGA, Arg-TGA) Nonsense mutation(The codon for encoding arginine isCGAAndAGA）。AP003 The design purpose is to be able to readPTC, and re-introduce arginine during the peptide chain generation process to restore the production of full-length proteins.Arg-TGA Is the most common nonsense mutation in human genetic diseases, accounting for21%-22%。
Video source: Alltrna
In preclinical models,AP003 Has been shown in by commonPTCThe ability to restore the expression of full-length proteins and downstream functional activity in disease environments. In addition, preclinical studies to date have shown,AP003The safety profile is based on oligonucleotides andLNPThe prior experience with this therapy is consistent, which provides support for its clinical evaluation.
IThe purpose of the clinical study is to evaluateAP003Safety and Pharmacokinetic Properties of Single-Dose Administration (Dose Escalation) in Healthy Volunteers.
AlltrnaIn 2018 Year by Flagship Pioneering Established, the company's platform utilizes artificial intelligence./Machine Learning (AI/ML) technology, used for developing and providing a variety of programmable molecules with broad therapeutic potential. As one of the earliest in the world to deploytRNAOne of the companies in the therapy field, in2021Year and2023Completed in different years5000Million USD Seed Round Financing and1.09Billion US dollarsBRound of financing.
"It is the first time that the modified tRNA Used for human trials, marking an important step forward in our development of new methods to treat genetic diseases by targeting the same mutation."Alltrna CMO Nerissa KreherDr. said.
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