Alltrna
tRNA Medicines for Stop Codon Disease
Alltrna is advancing a new class of genetic medicines based on the power of tRNA biology to universally treat Stop Codon Disease, which encompasses thousands of genetic diseases caused by a premature termination codon (PTC) mutation and affects approximately 30 million people worldwide.
Most genetic medicines are developed by focusing on each individual disease or gene—a slow and complex process for addressing the more than 6,000 rare genetic diseases. Leveraging the unique biology of tRNA, Alltrna is accelerating rare disease drug development and developing a single medicine with the potential to treat thousands of rare diseases that are driven by the same genetic mutation. Beyond tRNA, no other genetic medicine modality has yet to demonstrate this potential.
Across all rare genetic diseases and some cancers, PTCs change the genetic code for an amino acid to a premature “stop” codon that results in a shortened protein with no or altered biological activity. Alltrna is engineering tRNAs to recognize PTCs, insert the correct amino acid, and restore full-length protein production.
In May 2023, Alltrna presented first proof-of-concept data showing they can design, modify, and deliver tRNA molecules to correct PTC, independent of gene and mutation location, paving the way to create a universal therapeutic for Stop Codon Disease. These data demonstrate that the application of automated high-throughput sequence and ML-driven modification optimization through Alltrna’s unique platform can significantly increase engineered tRNA activity and PTC readthrough. In August 2023, Alltrna raised a $109 million Series B financing to advance the company’s platform and its first drug candidates towards the clinic.
