Bedford-based Homology Medicines, Inc. is making strides in transforming the lives of individuals affected by rare genetic diseases. By utilizing its proprietary platform, the genetic medicines company offers unique human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to facilitate the delivery of genetic medicines in vivo. Homology Medicines' AAVHSCs enable the precise targeting of disease-relevant tissues through a single injection. Disease-relevant tissues include the liver, central nervous system, peripheral nervous system, blood-brain-barrier, bone marrow, cardiac and skeletal muscle, and eye. The company's lead product candidate is HMI-102, which is undergoing Phase 2 pheNIX clinical trials as a gene therapy for phenylketonuria (PKU) treatment in adults. Homology Medicines is also in the process of developing HMI-103 to tackle PKU for pediatric patients, as well as HMI-202 to treat metachromatic leukodystrophy, HMI-203 for mucopolysaccharidosis type II, and HMI-104 for paroxysmal nocturnal hemoglobinuria treatment.
Homology Medicines's ticker is FIXX
The company's shares trade on the NASDAQ stock exchange
They are based in Bedford, Massachusetts
There are 51-200 employees working at Homology Medicines
It is https://www.homologymedicines.com/
Homology Medicines is in the Healthcare sector
Homology Medicines is in the Biotechnology industry
The following five companies are Homology Medicines's industry peers: