Alltrna Raises $109 Million to Advance tRNA Medicines for Stop Codon Disease

Rare Daily Staff

Alltrna closed on $109 million in a series B financing round to advance the company’s platform and first drug candidates toward the clinic for a first indication in Stop Codon Disease.

The financing round included company founder Flagship Pioneering and a number of top-tier investors.

Alltrna’s genetic medicines platform is based on the power of transfer RNA (tRNA) biology to universally treat Stop Codon Disease, which encompasses thousands of rare and common genetic diseases that stem from premature termination codons (PTC) 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. Approximately 10 percent of all people with a genetic disease have stop codon disease, representing approximately 30 million people worldwide. Alltrna is engineering tRNA medicines that can read these PTC mutations and deliver the desired amino acid, thereby restoring the production of the full-length protein.

“Since launch, we’ve gained unparalleled insights into tRNA biology and shown that we can systematically design and deliver tRNAs with universal readthrough of premature termination codons and restore full-length functional protein production in vivo,” said Michelle Werner, CEO of Alltrna and CEO-partner of Flagship Pioneering. “Since launch, we’ve gained unparalleled insights into tRNA biology and shown that we can systematically design and deliver tRNAs with universal readthrough of premature termination codons and restore full-length functional protein production in vivo. For the first time, we have the opportunity to universally treat stop codon disease, which encompasses thousands of rare and common human diseases driven by PTC mutations, giving millions of patients the chance at a treatment in years not lifetimes.”

tRNAs not only play a central role in the translation of mRNA into proteins but also are programmable molecules with a diverse biology of sequences and modifications. There exist more than 1034 tRNA sequences with more than 120 natural and synthetic modifications possible for each nucleotide, yielding the potential to generate more engineered, modified tRNA oligonucleotides than atoms in the universe.

Alltrna’s platform uniquely combines internal expertise and proprietary machine learning tools to harness the combinatorial potential of tRNA sequences and modifications that are key to structure, function, and stability. Alltrna recently presented data validating the potential of its platform to design, modify, produce, and deliver engineered, modified tRNA oligonucleotides with significantly increased potency and activity for the in vivo readthrough of PTC mutations to restore the production of full-length proteins, independent of gene and mutation location.

Photo: Michelle Werner, CEO of Alltrna and CEO-partner of Flagship Pioneering