Key information

🔎 Fund Overview

Homology Medicines, Inc. is a clinical-stage genetic medicines company focused on providing treatment for rare genetic diseases. The Company’s clinical programs include HMI-103, an investigational gene editing candidate in clinical development for the treatment of patients with phenylketonuria (PKU); HMI-203, an investigational gene therapy candidate in clinical development for the treatment of patients with mucopolysaccharidosis type II (MPS II), or Hunter syndrome; and HMI-102, an investigational gene therapy candidate in clinical development for the treatment of adult patients with PKU. Its lead GTx-mAb gene therapy candidate, HMI-104, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Its platform is designed to utilize human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs), to precisely and efficiently deliver single administration genetic medicines in vivo through a nuclease-free gene editing modality and gene therapy platform.