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Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting
- New Preclinical Data Demonstrated Ability to Re-Dose with AAVHSCs Across Viral Clades, Potentially Expanding Possibilities of Genetic Medicine Programs in
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[{"type":"text","content":"- New Preclinical Data Demonstrated Ability to Re-Dose with AAVHSCs Across Viral Clades, Potentially Expanding Possibilities of Genetic Medicine Programs in the Future - - Presented Methods to Accelerate Identification of Genomic Sites that Result in Improved Homologous Recombination-Based Gene Editing Efficiency - BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production. “Given the severity of PNH, which is a rare, acquired blood disorder, and the unmet need associated with chronically administered available therapies, HMI-104 could provide an important new one-time approach,” said Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “We are continuing to advance HMI-104 through IND-enabling studies as well as progress our GTx-mAb platform for ocular diseases, with potential for other indications with larger patient populations.” Homology also presented for the first time non-clinical data that showed the potential to re-dose liver-targeted AAVs, including the Company’s AAVHSCs, across different clades of viruses without suppressing or modulating the immune system. Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates. Highlights from Homology’s ASGCT 2023 Presentations HMI-104: GTx-mAb Development Candidate for PNHHomology presented results from two dose range-finding studies in the poster, “Preclinical Studies with HMI-104, an AAVHSC Vectorized C5 Monoclonal Antibody, for the Treatment of PNH.” In both NOD SCID and humanized liver murine models, a single intravenous (I.V.) dose of HMI-104 ...