Transfer RNAs are key to the protein-making process in cells, and some biotech companies are targeting these molecules as potential new treatments for disease.The latest such company is hc Bioscience, which is now in $24 million.
The Series A round, announced Wednesday, was led by a consortium that includes ARCH Venture Partners, Takeda Ventures and 8VC.
Many diseases are caused by dysfunctional proteins. In some cases, this dysfunction is because the protein is incomplete. Errors in a protein’s DNA blueprint cause genetic instructions to prematurely stop the protein-making process, resulting in protein truncation. This type of error is called a “nonsense mutation”. Such mutations are responsible for 10 to 15 percent of human disease, according to Cambridge, Massachusetts-based hC Bio.
Transfer RNA, or tRNA, is key to the protein manufacturing process, ensuring that key amino acids are correctly added to the protein as it is made. hC Bio’s technology, called PTCX, uses tRNA to suppress nonsense mutations in the genetic code, allowing the cell’s protein-making machinery to produce the appropriate full-length protein.
The startup also has a second technology, PTCX, to address diseases caused by missense mutations, in which changes in DNA sequence cause the wrong amino acid to be incorporated into proteins. PTCX technology marks disease-causing proteins for destruction by cells.
hC Bio President and CEO Leslie Williams and University of Iowa Professor of Molecular Physiology and Biophysics Christopher Ahern co-founded the startup. Ahern’s lab identified engineered tRNAs that could be used to process instructions that prematurely halt the protein-making process.The lab’s tRNA research is post In Nature Communications 2019.
“We are developing drugs that restore protein function to its intended state without editing the gene,” Williams said in a prepared statement. “Single tRNA therapies have the potential to work regardless of the gene or location of the mutation. potentially treat many diseases.”
Williams’ experience includes founding and serving as President and CEO of ImmusanT, a developer of therapeutic vaccines. Her new company did not specify which diseases it intends to treat with tRNA, but it joins several biotech companies developing treatments in this emerging class of genetic medicines.Cambridge last November Startup Alltna uses tRNA technology and $50 million launch from VC firm Flagship Pioneering. Similar to hC Bio’s PTCX technology, AlltRNA is designing tRNAs to treat diseases caused by nonsense mutations.
Another Cambridge biotech company, Tevard Biosciences, is developing RNA-modulating tRNA therapies to address nonsense mutations and haploinsufficiency, diseases in which one gene in a gene pair does not function properly resulting in insufficient levels of the protein. Tevard’s science is partly based on Ahern’s research.
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