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hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data from tRNA-based Protein Editing Platform

By: Newsfile

Boston, Massachusetts--(Newsfile Corp. - May 24, 2024) - 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 program in Duchenne muscular dystrophy (DMD) at the CureDuchenne FUTURES National Conference in Orlando, Fla.

To view the full announcement, including downloadable images, bios, and more, click here.

Key Takeaways:

  • Program is developing anticodon engineered tRNAs as a potential breakthrough treatment option for Duchenne muscular dystrophy (DMD) patients with shortened and nonfunctional dystrophin due to premature termination codons (PTCs)
  • Data from mouse model of DMD demonstrate restoration of full-length dystrophin in muscle cells
  • DMD program is currently in the early lead identification stage and joins hC Bioscience's growing pipeline, which also includes programs for severe hemophilia A and cancer

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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 the potential to treat many mutated genes using the same therapy. Our pipeline comprises a lead program for severe hemophilia A as well as programs for Duchenne muscular dystrophy and cancer. 


Justin Chen

Source: hC Bioscience

To view the source version of this press release, please visit

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