Transfer RNA (tRNA) is the essential, often overlooked, molecule that provides the critical translation of genetic code into the proteins that are the building blocks of life.
We are creating engineered tRNAs with the power to overcome disease-causing issues in the genetic code as proteins are made.
DNA is our genetic blueprint, sequestered in the nucleus of cells. Messenger RNA (mRNA) is the transcript of that blueprint, bringing instructions out of the nucleus. tRNA reads a specific three-letter nucleotide sequence of mRNA (a codon) and transfers the corresponding amino acid to the growing end of a polypeptide chain that will become a protein.
Errors in the DNA blueprint can cause disease. One type of error, called a “nonsense mutation” or “premature termination codon” (PTC), is responsible for 10-15% of all human genetic diseases. In these diseases, the codon for an amino acid has been changed to the code for “stop”, causing the polypeptide chain to terminate early and result in an incomplete and dysfunctional protein.
Our PTCX (“Patch”) platform uses engineered tRNAs to recognize nonsense mutations in mRNA transcripts and to suppress them by inserting an amino acid where it is needed, restoring the protein to full length and function. PTCX harnesses the unrealized power of tRNAs to fix the genetic message without changing the code itself.
hC Bio is simultaneously developing a second tRNA-based platform called SWTX (“Switch”) to target diseases caused by unwanted protein. This technology is designed to mark the disease-causing proteins for destruction.
Unlike gene therapy or gene editing, which must be customized for each mutation and patient, a single tRNA medicine has the potential to treat many diseases regardless of the gene or location of the mutation.