International Congress for Ataxia Research
Capsida Biotherapeutics to Present New Preclinical Data for Potential Best-in-Class Friedreich's Ataxia Next-Generation Gene Therapy
Capsida Biotherapeutics today announced new preclinical data for CAP-004, a potential best-in-class 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.
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.
Friedreich's ataxia is a rare hereditary disease with limited treatment options. It is caused by a mutation that reduces frataxin (FXN) protein expression, leading to progressive neurodegeneration, cardiac dysfunction, and sensory deterioration. CAP-004 is designed to target and address the key pathophysiological features of the disease by delivering therapeutically meaningful FXN expression in the CNS, cardiac tissues, and sensory regions.
In the study conducted in non-human primates (NHPs), results showed that CAP-004 demonstrated:
• Brain-wide biodistribution, achieving more than 80% transduction in key cell types, such as Purkinje cells in the cerebellum, neurons in the dentate nucleus, and motor neurons in the cortex and spinal cord.
• 100 times higher RNA expression compared with AAV9 in the cerebellum, motor cortex, and spinal cord.
• Significant cardiac tissue transduction, covering nearly 30% of the left ventricle tissue area.
• Bulk hFXN protein levels in treated NHPs were up to 8.2 times higher than endogenous levels in the motor cortex and 1.7 times higher in the left ventricle, highlighting the therapeutic potential in both CNS and cardiac regions.
• Potential in treating sensory vision loss, with significant RNA expression detected in the retina.
• Significant detargeting of the liver and other non-target tissues contributed to the favorable safety profile characterized by no adverse immunogenicity, clinical pathology, and histopathology findings.
"These preclinical data demonstrate CAP-004's potential to be a best-in-class gene therapy for Friedreich's ataxia, a devastating disease with few treatment options," said Peter Anastasiou, CEO of Capsida Biotherapeutics. "With CAP-004 delivered as a single IV injection, Capsida has the potential to address neurodegenerative, cardiovascular, and sensory components of this disease."
