CAP-004

CAP-004 is a next-generation, systemically administered gene therapy developed by Capsida Biotherapeutics for the treatment of Friedreich’s ataxia (FA), a rare hereditary neurodegenerative disease. It utilizes a proprietary engineered adeno-associated virus (AAV) capsid to deliver a functional copy of the frataxin (FXN) gene via intravenous infusion. The therapy is designed to target central nervous system (CNS), cardiac, and sensory tissues—key sites affected in FA—while minimizing off-target effects, particularly in the liver. Preclinical studies in non-human primates have demonstrated that CAP-004 achieves high levels of FXN expression in neurons, cardiomyocytes, and sensory cells, leading to increased production of mature frataxin protein and significant transduction efficiency across CNS and cardiac tissues. The goal is long-term restoration of cellular respiration and prevention of cell death in disease-relevant cell types with a single IV dose. As of late 2024/early 2025, CAP-004 is entering IND-enabling studies with plans for clinical development[1][2][3][4][5].