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

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

May 24, 2024 – Boston, Mass. — 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. 

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

Media Gallery

Related Bios

Leslie Williams
Co-Founder, President & CEO
Leslie J Williams, a 25-year biopharmaceutical industry veteran, is an experienced biotech CEO and board of directors’ member with demonstrated expertise in building companies, raising funds, and negotiating strategic collaborations.

Leslie is founding CEO, President & Director of hC Bioscience, Inc. an innovator and leader in protein editing with tRNA based therapeutics. She also serves on the Board of Directors for Ocular Therapeutix (OCUL), Windtree Therapeutics (WINT), and CSCRI (Coral Sea Clinical Research Institute). In addition, she serves on the Editorial Advisory Board of Life Science Leader, the Advisory Board of Life Science Cares and serves as an EIR at UVA and University of Iowa. Ms. Williams was formerly the founder and CEO of ImmusanT, developing immunotherapies for autoimmune diseases and was CEO at Ventaira Pharmaceuticals developing therapies using the lung as a portal for delivery. Williams prior pharmaceutical experience includes INO Therapeutics, Merck and GSK. She was also a venture partner at Battelle Ventures where she sourced and evaluated deals and assisted early-stage technology companies with strategy, management, business development and M&A.

Williams holds an MBA from Washington University, John Olin School of Business, and a B.S. degree with honors in nursing from the University of Iowa. Before entering the industry, she was a critical-care nurse at Duke University, Medical College of Virginia and at the University of Iowa.
View Full Bio>>
Jose Lora, Ph.D.
Chief Scientific Officer
Jose M. Lora is a seasoned biopharmaceutical leader who brings 20+ years’ experience in Research and Development across multiple modalities and therapeutic areas. Most recently, Jose served as Chief Scientific Officer at Intergalactic Therapeutics, a non-viral gene therapy company focusing on multiple areas with a major emphasis in Ophthalmology. At Intergalactic, Jose was responsible for all aspects of Research, Preclinical Development and Manufacturing, and his team was able to demonstrate unprecedented safe and durable expression in the retina of a very large gene, ABCA4. Prior to Intergalactic, Jose was Chief Scientific Officer at enGene, a clinical-stage non-viral gene therapy company focused on gene delivery to mucosal surfaces. At enGene, he and his team contributed to the conception, development and advancement of EG-70, a plasmid-based nanoparticle therapeutic for the treatment of bladder cancer, to Ph1/Ph2 clinical trials. Prior to enGene, Jose was Vice President of Research at Synlogic, a clinical-stage biopharmaceutical company dedicated to the application of synthetic biology to the discovery and development of therapies for metabolic and immune-mediated diseases, and cancer. At Synlogic, Jose built and led the Immunomodulation therapy area, and he led a multidisciplinary team to conceive, develop and advance SYNB1891, an engineered bacterial cell therapy designed to engage the STING pathway in solid tumors, to Ph1 clinical trials. Prior to Synlogic, he served in several biotech and pharmaceutical companies in positions of increasing impact and responsibility (Constellation -acquired by MorphoSys-, GlaxoSmithKline, Roche and Millennium -acquired by Takeda).

Jose received his PhD from the University of Seville, Spain, and was a postdoctoral researcher at Brown University and The University of Utah. He has authored numerous patent applications, as well as publications including papers in Journal of Experimental Medicine, PNAS, Nature, Nature Immunology, Nature Communications and Nature Reviews.
View Full Bio>>

Contacts