Homology Medicines, Inc., a genetic medicines company, focuses on translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. Its proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a range of genetic disorders. The company's various set of AAVHSCs allows company to target, through a single injection, a range of disease-relevant tissues, including the liver, central nervous system, bone marrow, lung, muscle, and eye in gene editing and gene therapy modalities. Its lead product candidate is HMI-102, a gene therapy for the treatment of phenylketonuria (PKU) in adults. The company also develops HMI-103 for the treatment of PKU in pediatric patients; and HMI-202 to treat metachromatic leukodystrophy. Homology Medicines, Inc. was founded in 2015 and is based in Bedford, Massachusetts.