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     138  0 Kommentare Homology Medicines Presents New Data Characterizing AAVHSCs as Potential Gene Therapies for Nervous System Disorders

    - Data Demonstrate AAVHSCs Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier, Produced Normal Levels of Human Protein and Reduced Key Biochemical Markers of Disease in Preclinical Models -

    BEDFORD, Mass., Feb. 13, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today presentations demonstrating that its proprietary adeno-associated virus vectors derived from hematopoietic stem cells (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in preclinical models. Additionally, in the oral presentation on metachromatic leukodystrophy (MLD), a lysosomal storage disorder caused by mutations in the ARSA gene, Homology’s gene therapy candidate HMI-202 reduced key biomarkers of disease and produced normal levels of human ARSA protein in the murine model of MLD. These data presented at the WORLDSymposium 2020 Conference support the further development of AAVHSC-based gene therapies for diseases of the nervous system.

    “The data presented this week build upon our previously published AAVHSC biodistribution data to include characterization of 11 of our AAVHSC vectors and showed that all of them crossed the blood-brain-barrier and blood-nerve-barrier in NHPs,” stated Albert Seymour, Ph.D., Chief Scientific Officer. “This enables us to choose the most suitable vector for different diseases, just as we have done in selecting HMI-202 for our MLD program, which is in IND-enabling studies. We look forward to pursuing additional opportunities to develop gene therapies based on our AAVHSCs for the treatment of neurological disorders as well as other rare diseases.”

    In the poster presentation, “AAVHSC Characterization for Developing Treatments for Human Genetic Diseases of the Nervous System,” 11 of Homology’s AAVHSCs were evaluated and, following a single I.V. administration in non-human primates (NHPs), demonstrated broad tissue tropism (entering the cells) in the central and peripheral nervous systems. Specifically, AAVHSCs:

    • Crossed the blood-brain-barrier and blood-nerve-barrier.
    • Demonstrated various levels of tropism within the spinocerebellar pathway, suggesting individual AAVHSCs may be preferentially chosen based on disease biology.
    • Showed broad tissue tropism, making them amenable for potential therapeutic applications.

    In the oral presentation, “HMI-202: Investigational Gene Therapy for Treatment of Metachromatic Leukodystrophy (MLD),” a single I.V. administration of HMI-202 was evaluated in the MLD ARSA knockout murine model and in NHPs. A single administration of HMI-202 resulted in:

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    Homology Medicines Presents New Data Characterizing AAVHSCs as Potential Gene Therapies for Nervous System Disorders - Data Demonstrate AAVHSCs Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier, Produced Normal Levels of Human Protein and Reduced Key Biochemical Markers of Disease in Preclinical Models - BEDFORD, Mass., Feb. 13, 2020 (GLOBE NEWSWIRE) - …