hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA

April 23, 2024 at 07:00 AM EDT

Lead program in severe hemophilia A aims to establish anticodon engineered tRNAs as breakthrough treatment option for 20 percent of patients with premature termination codon (PTC)-driven factor VIII deficiency

Preclinical protein editing data demonstrate restoration of full-length factor VIII despite PTC in open reading frame

Hemophilia A program aims to demonstrate proof of concept for a gene-agnostic approach across a wide range of diseases

hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced preclinical data supporting its lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain.

Jose Lora, Ph.D., Chief Science Officer of hC Bioscience, unveiled the company’s first development candidate, HCB-101, an anticodon engineered tRNA designed to suppress nonsense mutations. HCB-101 is delivered as a lipid nanoparticle to target the liver, the organ where Factor VIII is produced. The data presented demonstrate successful targeting of the liver in mice as well as successful in vitro production of full-length, functional factor VIII despite the presence of a premature termination codon (PTC) that would otherwise result in a truncated nonfunctional protein. This 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’re excited to develop tRNA-based protein editing as a potential new breakthrough treatment option for patients with severe hemophilia A. Our lead program is on track to rapidly move into the clinic, where we hope to establish our novel protein editing approach as an easily adaptable modality across a broad spectrum of genetically defined diseases,” said Leslie Williams, CEO of hC Bioscience. “Because our therapies are gene agnostic, the engineered tRNA that reads through PTCs in hemophilia will recognize the same PTC in the context of hundreds of other genetic disorders as well. We see tRNAs as not just a novel modality, but a powerful, universal drug development platform that expands the potential of genomic medicine to improve the lives of patients.”

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. The company is holding a clinical advisory board meeting in conjunction with the World Federation of Hemophilia 2024 World Congress.

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.