AVTS-001

AVTS-001 is an AAV (adeno-associated virus)-based gene therapy developed by Aevitas Therapeutics for the treatment of complement-mediated diseases. The therapy is designed to restore lasting production of regulatory proteins through gene transference[1]. It represents an investigational approach to address diseases involving complement dysregulation, including age-related macular degeneration, hemolytic uremic syndrome, and paroxysmal nocturnal hemoglobinuria[1]. The program was acquired by 4D Molecular Therapeutics in 2023[1]. Previously, the asset was part of Fortress Biotech's portfolio through their majority ownership of Aevitas[11].