RARE Daily

hC Bioscience Reports In Vivo Data for tRNA-based Editing Program in DMD

May 24, 2024

Rare Daily Staff

hC Bioscience reported proof-of-concept preclinical data that it said supports its approach of using tRNA-based protein editing to treat the rare and fatal neuromuscular disease Duchenne muscular dystrophy when caused by an underlying nonsense mutation.

The company announced its data at the CureDuchenne FUTURES National Conference in Orlando, Florida. CureDuchenne, through its Cure Duchenne Ventures arm, is an investor in hC Bioscience.

Jose Lora, hc Bioscience’s chief scientific officer of hC Bioscience, presented the proof-of-concept mouse data demonstrating that delivery of anticodon engineered tRNAs to muscle cells restores production of full-length, native dystrophin despite the presence of a premature termination codon that would otherwise result in a truncated protein.

Premature termination codon’s account for approximately 26 percent of DMD cases.

“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.”

hC Bioscience’s DMD program is currently in the early lead identification stage.

Photo: Jose Lora, hc Bioscience’s chief scientific officer of hC Bioscience

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