Gene-editing via homologous recombination
HMI-103 Clin1 phenylketonuria; HMI-203 Clin1 MPS II; HMI-204 Clin0 metachromatic leukodystrophy; HMI-104 Clin0 paroxysmal nocturnal hemoglobinuria; 2 undisclosed programs Clin0 eye condition, undisclosed
Homology Medicines is translating gene editing and gene therapy technologies into novel treatments. Genetic variants drive many rare and debilitating diseases: current treatments only slow progression instead of treating the root cause. Homology is leveraging their genetic medicines platform to develop one-time gene therapies and gene editing treatments. The platform is based on the discovery of adeno-associated viruses found inside human stem cells that can deliver genetic material throughout the body, including into the brain. Homology’s pipeline includes candidates in clinical trials such as HMI-102, a gene therapy for adult phenylketonuria.