hC Bioscience Showcases Hemophilia A Preclinical Data at ASGCT

hC Bioscience, a pioneering biopharmaceutical firm based in Boston, unveiled promising preclinical data at the American Society of Gene and Cell Therapy Annual Meeting in Baltimore. The data pertains to their lead program targeting severe hemophilia A, a genetic disorder. This novel approach hinges on tRNA-based protein editing, a technology that could revolutionize treatment for genetic diseases.

Dr. Suchul Jang, Senior Director of Biology at hC Bioscience, presented findings that highlighted the potential of HCB-101, an anticodon engineered (ACE) tRNA. This innovative tRNA can correct premature termination codons (PTC) in the Factor VIII gene, a mutation that typically produces a truncated and non-functional protein. Specifically, their preclinical data demonstrated the successful restoration of full-length Factor VIII protein in vitro using HCB-101. Additionally, the data revealed effective delivery of this tRNA via lipid nanoparticles to target liver cells in mouse models. This approach could potentially be applied to about 20 percent of severe hemophilia A cases and has broader implications for other conditions caused by nonsense mutations.

Leslie Williams, CEO of hC Bioscience, emphasized the transformative potential of their tRNA-based protein editing technology. Williams noted that hemophilia A is just the initial target, but the same technology could be adapted to treat a wide array of liver-based disorders, including various bleeding and metabolic diseases, as well as conditions like Duchenne muscular dystrophy and cancer.

One of the key advantages of this tRNA protein editing strategy is its non-intrusive nature. Unlike traditional gene therapy methods that alter the genetic material itself, tRNA editing reverses the effects of disease-causing mutations without modifying the DNA. This makes the treatment reversible and less likely to interfere with normal cellular functions. Williams expressed optimism about the forthcoming clinical trials, hoping to position their approach as a groundbreaking treatment for hemophilia A and establish proof of concept for their broader platform.

To advance their lead program, hC Bioscience is preparing IND-enabling studies, aiming to initiate a Phase 1 clinical trial for severe hemophilia A by 2025. These efforts underscore the company's commitment to developing tRNA-based therapeutics that can address a diverse range of genetically defined diseases and cancer.

hC Bioscience is focused on creating first-in-class tRNA-based treatments that correct nonsense mutations, which otherwise lead to nonfunctional proteins. This gene-agnostic technique forms the basis of a universal drug platform capable of targeting multiple mutated genes with a single therapy. Their lead program aims to restore the production of functional Factor VIII protein in patients suffering from severe hemophilia A, offering a new hope for those affected by this debilitating condition.