WuXi AppTec Content Team Editor
hC Bioscience announced today that it has closed a $24 million Series A financing to develop potential tRNA-based “first-in-class” therapies for a variety of genetic diseases. Unlike other personalized therapies that target mRNA, this innovative tRNA-based therapy has the potential to treat multiple diseases with one therapy.
The company is advancing two complementary platforms. The platform, called PTCX, is designed to directly restore protein function when “nonsense mutations” lead to premature stop codons, causing protein dysfunction. This problem is responsible for 10-15% of human disease. The tRNA synthesized by this platform is able to read through premature stop codons, allowing the production of suitable full-length proteins.
▲Mechanism of PTCX technology platform (picture source: hC Bioscience official website)
The company is also developing a second tRNA-based technology platform called SWTX, which is used to treat diseases caused by missense mutations, production of too much protein, or unwanted protein disease. The technology aims to tag these disease-causing proteins so they can be degraded by the cell’s protein degradation system.
▲The mechanism of action of the SWTX platform (Image source: hC Bioscience’s official website)
“We are creating drugs that restore protein function without editing the gene,” said Ms. Leslie J. Williams, President and CEO of hC Bioscience, “whatever the gene is. , or where the genetic mutation occurs, a tRNA therapy has the potential to treat a variety of diseases.”