Homology Medicines Presents New Data Characterizing AAVHSCs as Potential Gene Therapies for Nervous System Disorders
Homology Medicines, Inc. (FIXX)
Last homology medicines, inc. earnings: 3/12 04:10 pm
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Source: GlobeNewswire
- 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.
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