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Homology Medicines Announces Upcoming Presentations Highlighting its Genetic Medicines Platform, including New Data from IND-Enabling Studies with GTx-mAb Development Candidate HMI-104 for PNH, at ASGCT Annual Meeting
- First Preclinical Data Sets Demonstrating Potential to Re-Dose with AAVHSCs and to Identify Genomic Sites with Improved Gene Editing Integration Efficiency
About this update from Q32 Bio Inc.
[{"type":"text","content":"- First Preclinical Data Sets Demonstrating Potential to Re-Dose with AAVHSCs and to Identify Genomic Sites with Improved Gene Editing Integration Efficiency - BEDFORD, Mass., May 03, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the first presentation of results from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of functional C5 monoclonal antibody (C5mAb) levels in multiple preclinical models. Homology will also present for the first time non-clinical data that show the potential for AAVHSC re-dosing across different clades of viruses. These presentations, alongside additional data supporting the Company’s gene editing and gene therapy programs, will be featured at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting from May 16-20, 2023. “These new data to be presented during ASGCT with our GTx-mAb development candidate, HMI-104, support its development as a one-time treatment for the rare, acquired disorder PNH, with potential for the platform to address other complement-mediated disorders,” said Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “We also look forward to unveiling data that demonstrate the ability to re-dose AAVHSCs across different virus clades, which is of particular importance as the field continues to focus on AAV re-dosing strategies. Additional presentations will highlight work across our AAVHSC platform, including methods to further characterize genomic targets with the highest likelihood of homologous recombination-based gene editing, as we work toward our goal of delivering best-in-class gene editing therapies.”Homology’s ASGCT 2023 presentations will include: HMI-104: GTx-mAb Development Candidate for PNHPreclinical Studies with HMI-104, an AAVHSC Vectorized C5 Monoclonal Antibody, for the Treatment of PNH Wednesday, May 17 at 12:00 p.m. PTAbstract # 386 AAVHSC PlatformOcular Biodistribution of AAVHSCs Across Species and Routes of Administration Wednesday, May 17 at 12:00 p.m. PTAbstract # 376 Immunosuppression Regimen Used in Ongoing Clinical Trials for PKU and MPS IITargeted Approach to Immunosuppression wit...