Homology Medicines Inc

NASDAQ:QTTB

Homology Medicines, Inc. is a clinical-stage genetic medicines company, which engages in the design and development of treatments to address rare diseases at the genetic level. The company is headquartered in Bedford, Massachusetts and currently employs 224 full-time employees. The company went IPO on 2018-03-28. The Company’s clinical programs include HMI-102, is an investigational gene therapy candidate in clinical development for the treatment of adult patients with phenylketonuria (PKU); HMI-103, which is an investigational gene editing candidate in clinical development for the treatment of patients with PKU, and HMI-203, which is an investigational gene therapy candidate in clinical development for the treatment of patients with mucopolysaccharidosis type II (MPS II), or Hunter syndrome. Additionally, the Company is developing a gene therapy candidate, HMI-104, from its GTx-mAb platform for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), and conducting research in other diseases, including metachromatic leukodystrophy (MLD). Its platform is designed to utilize human hematopoietic stem cell derived adeno-associated virus vectors.