hC Bioscience Announces Program in Duchenne Muscular Dystrophy and Reports In Vivo Data From tRNA-based Protein Editing Platform
2024年5月24日 - 8:00PM
ビジネスワイヤ(英語)
- Program is developing anticodon engineered
tRNAs as 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
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.
Jose Lora, Ph.D., Chief Scientific Officer of hC Bioscience,
presented proof-of-concept murine data demonstrating that delivery
of 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 that affect
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.
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.
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Justin Chen jchen@tenbridgecommunications.com