Description

Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic 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 for various 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, peripheral nervous system, bone marrow, cardiac and skeletal muscle, and eye. Its lead product candidate is HMI-102, which is in Phase 2 pheNIX clinical trial, 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; HMI-202 to treat metachromatic leukodystrophy; and HMI-203 for the treatment of mucopolysaccharidosis type II, as well as HMI-104 for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.