Homology Medicines
Company Type: Therapeutics
Main focus: Using a novel class of AAVs (AAVHSCs) and nuclease-free genome editing to treat a wide range of genetic diseases. The company also has gene therapy and GTx-mAb platforms.
Company stage: Clinical
Diseases: Diverse rare diseases
Genome-editing tool: nuclease-free
Funding stage: Series B
Location: Lexington, Massachusetts, United States
Website: https://www.homologymedicines.com/
Pipeline: https://www.homologymedicines.com/pipeline/
Partners: Oxford Biomedica
Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from rare diseases by addressing the underlying cause of the disease. The company's clinical programs include HMI-103, a gene-editing candidate for phenylketonuria (PKU); HMI-203, an investigational gene therapy for Hunter syndrome; and HMI-102, an investigational gene therapy for adults with phenylketonuria. The company's proprietary platform is designed to utilise its family of 15 human haematopoietic stem cell-derived adeno-associated virus (AAVHSCs) vectors to precisely and efficiently deliver genetic medicines in vivo through a gene therapy or nuclease-free gene-editing modality, as well as to deliver one-time gene therapy to produce antibodies throughout the body through the GTx-mAb platform.