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Homology Medicines, Inc. is a clinical-stage genetic medicines company. The Company is focused on transforming the lives of patients suffering from rare genetic diseases with unmet medical needs by addressing the underlying cause of the disease. Its platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs), to deliver single administration genetic medicines in vivo through a nuclease-free gene editing modality, gene therapy, or gene therapy to express antibodies platform (GTx-mAb), which is designed to produce antibodies throughout the body. The Company has gene editing and gene therapy clinical-stage programs in phenylketonuria (PKU) and Hunter syndrome (MPS II), a preclinical pipeline that includes a gene therapy candidate for metachromatic leukodystrophy and a GTx-mAb (vectorized antibody) candidate for paroxysmal nocturnal hemoglobinuria, as well as intellectual property on its family of 15 AAVHSCs.