hC Bioscience Announces Lead Program in Hemophilia and Reports Positive Preclinical Data on Novel Protein Editing Approach Using Anticodon Engineered tRNA

Key Takeaways

Lead program in severe hemophilia A aims to establish anticodon engineered tRNAs as breakthrough treatment option for 20 percent of patients with premature termination codon (PTC)-driven factor VIII deficiency
Preclinical protein editing data demonstrate restoration of full-length factor VIII despite PTC in open reading frame
Hemophilia A program aims to demonstrate proof of concept for agene-agnostic approach across a wide range of diseases

April 23, 2024 – Boston, Mass. — hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNAbased protein editing, today announced preclinical data supporting its lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain.

Jose Lora, Ph.D., Chief Scientific Officer of hC Bioscience, unveiled the company’s first development candidate, HCB-101, an anticodon engineered tRNA designed to suppress nonsense mutations. HCB-101 is delivered as a lipid nanoparticle to target the liver, the organ where Factor VIII is produced. The data presented demonstrate successful targeting of the liver in mice as well as successful in vitro production of full-length, functional factor VIII despite the presence of a premature termination codon (PTC) that would otherwise result in a truncated nonfunctional protein. This approach has the potential for application in about 20 percent of severe hemophilia A cases and could be extended across a broad spectrum of other diseases caused by nonsense mutations.

“We’re excited to develop tRNA-based protein editing as a potential new breakthrough treatment option for patients with severe hemophilia A. Our lead program is on track to rapidly move into the clinic, where we hope to establish our novel protein editing approach as an easily adaptable modality across a broad spectrum of genetically defined diseases,” said Leslie Williams, CEO of hC Bioscience. “Because our therapies are gene agnostic, the engineered tRNA that reads through PTCs in hemophilia will recognize the same PTC in the context of hundreds of other genetic disorders as well. We see tRNAs as not just a novel modality, but a powerful, universal drug development platform that expands the potential of genomic medicine to improve the lives of patients.”

hC Bioscience will build on these preclinical data through IND-enabling studies with the goal of enrolling a Phase 1 clinical trial for severe hemophilia A in 2025. The company is holding a clinical advisory board meeting in conjunction with the World Federation of Hemophilia 2024 World Congress.

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 potential to treat many mutated genes using the same therapy. Our lead program is directed at restoring full-length and functional FVIII protein in people with severe hemophilia A.

Key Takeaways

Lead program in severe hemophilia A aims to establish anticodon engineered tRNAs as breakthrough treatment option for 20 percent of patients with premature termination codon (PTC)-driven factor VIII deficiency
Preclinical protein editing data demonstrate restoration of full-length factor VIII despite PTC in open reading frame
Hemophilia A program aims to demonstrate proof of concept for agene-agnostic approach across a wide range of diseases

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1) In healthy RNA, the ribosome moves seamlessly along the mRNA chain with tRNA shuttling amino acids to produce a functional, full-length protein. 2) If a mutation results in a premature termination codon, the body can be deprived of a protein essential for preventing disease. 3) Therapeutic PTCX tRNA recognizes premature termination codons, overwrites, and repairs the PTC by adding the correct amino acid and allowing for protein synthesis to continue to completion. Available for use by m

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Our lead program is on track to rapidly move into the clinic, where we hope to establish our novel protein editing approach as an easily adaptable ...

Leslie Williams — Co-Founder, President and CEO

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.

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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.

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Justin Chen

jchen@tenbridgecommunications.com

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