HCB101

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 a gene-agnostic approach across a wide range of diseases BOSTON--(BUSINESS WIRE)-- 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 lead program in severe hemophilia A at the World Federation of Hemophilia 2024 World Congress in Madrid, Spain. Jose Lora, Ph.D., Chief Science 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.

SAN FRANCISCO, CA and TAIPEI, TAIWAN / ACCESSWIRE / December 1, 2023 / HanchorBio Inc., a global clinical-stage biotechnology company focusing on the discovery and development of innovative immuno-biomedicines to treat a wide variety of patients suffering from hard-to-treat solid tumors or hematological malignancies, today announced the acceptance of poster presentation at the European Society for Medical Oncology Immuno-Oncology (ESMO-IO) 2023 Annual Congress, taking place in Geneva, Switzerland and online from December 6-8, 2023. Details of the presentation are as follows. The full abstracts will be made available on the ESMO website on Thursday, 30 November 2023 at 00:05 CET. Title: An Engineered Ligand-Trap Biologic Targeting the CD47 Signaling Pathway for Cancer Treatment with Superb Efficacy and Safety Profiles Presenter: Sean Juo, Ph.D., President and Chief Scientific Officer of HanchorBio Location: Palexpo, Geneva, Switzerland Presentation Number: #10951 Date and Time: Thursday, 7 December 2023 at 12:00-13:00 CET Highlights from the Preclinical Data- Key findings from the preclinical studies of HCB101 targeting the SIRPa- CD47 pathway include: HCB101 triggered strong phagocytic reactions against tumor cells but not red blood cells; HCB101 consistently showed excellent efficacy against hematological and solid malignancies in 14 human tumor xenograft models; Quantitative RNA transcriptional analysis indicated that HCB101 triggered drastic changes in gene expression inside the tumor compared to other competing molecules, suggesting a unique MOA underlying HCB101's superior efficacy; No adverse reaction was observed in cynomolgus monkey toxicology studies, indicating a good safety profile; HCB101 is currently being evaluated in a multi-regional, multi-center clinical study (NCT05892718) in the United States and Taiwan. About HCB101 Discovered through structure-guided protein design which was further supplemented with relevant screening technologies, HCB101 is an engineered extracellular IgV domain of SIRPα fused to the Fc region of IgG4. HCB101 triggers potent phagocytic activity of macrophages by blocking the "do not eat" signal between macrophages and the tumor cells. Based on the extensive in-vitro and in-vivo preclinical data, HCB101 is potentially a much safer and more potent biologic than the anti-CD47 monoclonal antibodies and other SIRPα fusion proteins currently being investigated in clinical trials. Furthermore, quantitative RNA transcriptional analysis indicated that HCB101 triggered distinct gene expression profiles inside the tumor and in the tumor microenvironment compared to other relevant clinical candidates, suggesting that HCB101 might exhibit unique mechanisms of action. With IND clearance from the U.S. FDA and Taiwan FDA, HCB101 is currently being investigated in a multi-regional, multi-center, open-label, dose-finding clinical trial (NCT05892718) of adults with advanced solid tumors or relapsed and refractory non-Hodgkin lymphoma in the United States and Taiwan. Using HCB101 as the foundation, our multi-functional Fc-based designer biologics (FBDB™) platform pipeline includes other immune-modulating molecules targeting both adaptive and innate immunity to provide novel and efficacious treatments to patients suffering from difficult-to-treat cancers and other debilitating diseases. About HanchorBio Based in Taipei, Shanghai, and the San Francisco Bay Area, HanchorBio is a global clinical-stage biotechnology company focusing on immuno-oncology. The Company is led by an experienced team of pharmaceutical industry veterans with a proven track record of success in biologics discovery and global development to transcend current cancer therapies. Committed to reactivating the immune system to fight against diseases, the proprietary Fc-based designer biologics (FBDB™) platform enables unique biologics with diverse multi-targeting modalities to unleash both innate and adaptive immunity to overcome the current inadequacies of anti-PD1/L1 therapies. The FBDB™ platform has successfully delivered proof-of-concept data in several in vivo tumor animal models. By making breakthroughs in multi-functional innovative molecular configurations in R&D and improving the manufacturing process in CMC, HanchorBio develops transformative medicines to address unmet medical needs. For more information, please visit HanchorBio or follow us on LinkedIn Contact Information Scott Liu Founder, Chairman and CEO scott_liu@hanchorbio.com Yuehua Cong Chief Business Officer yuehua_cong@hanchorbio.com SOURCE: HanchorBio Inc. View source version on accesswire.com: