HMI-104

HMI-104 is a preclinical **gene therapy-based monoclonal antibody** candidate from **Homology Medicines** for **paroxysmal nocturnal hemoglobinuria**. The program uses an **AAVHSC** vector to deliver a transgene encoding an **anti-C5 monoclonal antibody**, with the goal of enabling the liver to produce sustained systemic levels of the antibody after a **single intravenous administration**. By functionally inhibiting **complement component C5**, HMI-104 is intended to block terminal complement activation and reduce complement-mediated hemolysis in PNH. It is best described as a **vectorized antibody** or **GTx-mAb** program that combines features of gene therapy and biologic anti-complement therapy.