Homology Medicines Presents New Data Characterizing AAVHSCs as Potential Gene Therapies for Nervous System Disorders - Seite 2

• Durable and high levels (­≥100%) of human ARSA (hARSA) activity in the central nervous system (CNS) of the MLD murine model, with hARSA detected in all cell types of the nervous system.
• Reductions in key biochemical markers of MLD, including a reduction in LAMP-1 accumulation and a significant decrease in brain sulfatide levels in the murine model.
• Presence of hARSA in the peripheral nervous system and CNS in NHPs, suggesting that HMI-202 crossed the blood-brain-barrier and blood-nerve-barrier.

About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder caused by mutations in the ARSA gene. ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within five to ten years after onset.

About Homology Medicines, Inc.
Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing, and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.