- Preclinical data establish successful protein editing in vitro to overwrite premature termination codon (PTC) in Factor VIII gene
- In vivo data show successful delivery to target cells in liver
- Company readying IND-enabling studies to prepare for clinical trials in severe hemophilia A
hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today presented preclinical data supporting its lead program in severe hemophilia A at the American Society of Gene and Cell Therapy Annual Meeting in Baltimore, Md.
Suchul Jang, Ph.D., Senior Director, Biology, presented data demonstrating that HCB-101, an anticodon engineered (ACE) tRNA, restores production of full-length Factor VIII protein in vitro despite the presence of a premature termination codon (PTC) that would otherwise result in a truncated, non-functional protein. The data also show that HCB-101 can be delivered successfully via a lipid nanoparticle to target cells in the livers of mice. This tRNA-based protein editing approach has the potential for application in about 20 percent of severe hemophilia A cases and could be extended across a broad spectrum of other diseases caused by nonsense mutations.
“We see hemophilia A as the first of many diseases that could be addressed using our tRNA-based protein editing technology, which has broad potential to functionally undo the effects of any disease-causing nonsense mutation. This approach could be used to treat numerous other liver-based disorders beyond hemophilia A, such as an array of bleeding and metabolic disorders, as well as diseases such as Duchenne muscular dystrophy and cancer,” said Leslie Williams, CEO of hC Bioscience. “tRNA protein editing has multiple advantages, including the ability to tackle genetic diseases without affecting the genetic material itself, making it reversible and allowing the cell machinery to act normally. Our lead program is on track to move into the clinic, where we hope to establish it as a promising new treatment option for patients with hemophilia A and as clinical proof of concept for our innovative platform.”
hC Bioscience will build on these preclinical data through IND-enabling studies with the goal of enrolling a Phase 1 clinical trial for severe hemophilia A in 2025.
About hC Bioscience, Inc.
hC Bioscience is dedicated to improving the lives of patients through the development of first-in-class tRNA-based therapeutics that address a broad spectrum of genetically defined diseases and cancer. Our anticodon engineered tRNAs overwrite nonsense mutations that would otherwise result in truncated, nonfunctional proteins. This gene-agnostic approach is the foundation for a universal drug platform with potential to treat many mutated genes using the same therapy. Our lead program is directed at restoring full-length and functional FVIII protein in people with severe hemophilia A.
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