Capsida Biotherapeutics (“Capsida”) today announced new preclinical data for CAP-004, a systemically administered next-generation gene therapy for the treatment of Friedreich’s ataxia. CAP-004 is Capsida’s third wholly owned program and utilizes one of the company’s proprietary engineered capsids to target central nervous system (CNS), cardiac, and sensory manifestations of the disease while limiting effects in off-target tissues, especially the liver. CAP-004 is now entering Investigational New Drug (IND) application enabling studies. Learn more about the stages of drug development.
Data highlighting the therapeutic potential of CAP-004 will be presented in a late-breaking poster presentation at the International Congress for Ataxia Research (ICAR), being held November 12-15, 2024 in London, UK.