Pharma Focus America

Hc Bioscience Introduces Program for Duchenne Muscular Dystrophy and Releases In Vivo Results of tRNA-based Protein Editing Platform

Monday, May 27, 2024

HC Bioscience, a biopharmaceutical company pioneering a novel approach to treating genetic diseases through tRNA-based protein editing, presented preclinical data supporting its program in Duchenne muscular dystrophy (DMD) at the CureDuchenne FUTURES National Conference in Orlando, Fla.

HC Bioscience, showcased proof-of-concept murine data demonstrating that delivering anticodon engineered (ACE) tRNAs to muscle cells restores production of full-length, native dystrophin despite the presence of a premature termination codon (PTC) that would otherwise result in a truncated protein. In DMD, a rare, fatal, progressive neuromuscular disease caused by mutations to dystrophin affecting about 300,000 males worldwide, PTCs account for approximately 26 percent of cases.

“We are grateful for the opportunity to share these results with the DMD community as we explore the potential of tRNA-based protein editing. No other treatment, approved or investigational, restores production of full-length dystrophin, and tRNA-based protein editing achieves that goal without altering the genome,” said Leslie Williams, CEO of hC Bioscience. “This rapidly emerging modality has the potential to be a new breakthrough therapeutic for about one in four people affected by DMD. We see protein editing as a key innovation for advancing patient care, and our goal is to work with families, physicians, and clinical researchers to realize its full promise.”

HC Bioscience’s DMD program is currently in the early lead identification stage and is supported by an investment from Cure Duchenne Ventures, the strategic investment arm of CureDuchenne, a global nonprofit committed to finding and funding a cure for Duchenne muscular dystrophy.



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