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Homology Medicines Announces Presentations on its In Vivo Gene Therapy and Gene Editing Programs and Commercial Manufacturing Platform at the American Society of Gene & Cell Therapy Annual Meeting
- Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editing for PKU - - Manufacturing Enhancements Led to Improved Productivity,
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[{"type":"text","content":"- Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editing for PKU -\n - Manufacturing Enhancements Led to Improved Productivity, Quality and Scalability of Commercial Process, Confirmed in 2,000L Bioreactor - - Data Highlight Unique Characteristics of AAVHSC Genetic Medicines Platform - BEDFORD, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of data at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. Among Homology’s seven presentations are data from its in vivo nuclease-free gene editing program for phenylketonuria (PKU) and in vivo gene therapy program for metachromatic leukodystrophy (MLD), both of which are in IND-enabling studies. Presentations also focus on the Company’s commercial manufacturing platform, as well as data on the differentiating characteristics of Homology’s family of AAVHSC vectors, particularly when compared to other AAVs, which highlight the potential of the Company’s dual gene therapy and editing platform. “Homology has made substantial progress in understanding the unique properties of our AAVHSC-based technology and this enables us to move our dual genetic medicines platform forward to develop potential treatments, or cures, for patients,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “We are pleased to share data here that describe the molecular methods we have developed to characterize in vivo, nuclease-free gene editing efficiency and precision at the DNA level. Additional data from our in vivo MLD gene therapy program demonstrates the impact on key biomarkers in two species, as well as the durability of effect in the murine model of disease with data out to 52 weeks. Underpinning all our programs is our internal GMP process and manufacturing capabilities, where we have now confirmed our commercial HEK293 suspension platform at the 2,000-liter scale, bringing our total internal capacity to 3,500 liters. Additionally, we are presenting data showing improved AAVHSC packaging as compared to the non-Clade F vector AAV5.” Highlights from Homology’s 2020 ASGCT Presentations The presentation, “Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells using AAVHSC15, a New AAV Derived from Hematopoietic St...