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Homology Medicines Announces Upcoming Presentation on Optimized In Vivo Gene Editing Candidate HMI-103 with First Details of Unique Mechanism of Action at ASGCT Annual Meeting
- Multiple Presentations Feature HMI-103 Phase 1 Investigational Therapy for PKU, Including Genome-Wide Integration Assays to Confirm No Off-Target Editing -
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[{"type":"text","content":"- Multiple Presentations Feature HMI-103 Phase 1 Investigational Therapy for PKU, Including Genome-Wide Integration Assays to Confirm No Off-Target Editing - - New Data from GTx-mAb Program Support Potential to Target Many Complement-Related Disorders - - Presentations Feature the Discovery and Characterization of a Non-Liver-Tropic Capsid and Other Distinct Properties of Homology’s Family of 15 Naturally Derived AAVHSCs - - Symposium to be Held on May 18, 2022 at 7:30 a.m. ET - BEDFORD, Mass., May 02, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that the first presentation detailing the optimization and mechanism of action of its HMI-103 nuclease-free gene editing candidate for phenylketonuria (PKU) will take place during the American Society for Gene & Cell Therapy (ASGCT) 25th Annual Meeting May 16-19, 2022. The data to be presented, which include a genome-wide assay that detects on- and off-target gene integration, supported the initiation of the pheEDIT trial evaluating HMI-103 for PKU, the first gene editing study for this disease. New information from the Company’s GTx-mAb program, including data that further characterize the expression of C5 antibodies from AAVHSCs, will also be shared. Additionally, details on Homology’s family of 15 adeno-associated viruses derived from human hematopoietic stem cells (AAVHSCs) will be presented, including one demonstrating low tropism to the liver, and the unique properties that make them amenable to developing treatments for many genetic disorders. “Homology has focused on scientific and clinical innovation with the translation of our in vivo gene therapy and gene editing platform into one-time product candidates, and we believe that our approach to optimize nuclease-free gene editing to be unveiled at ASGCT is further evidence of our leadership in developing genetic medicines,” said Albert Seymour, Ph.D., President and Chief Scientific Officer of Homology Medicines. “Our team designed assays to ensure that we can scan the entire genome to detect on- and off-target events, and the preclinical data we plan to share confirm no evidence of off-target integration or unwanted mutations. Beyond the data backing our clinical programs and pipeline, we will report structural and functional analyses of our AAVHSCs including a nov...