Capsida’s AAV CAP-004 Gene Therapy Shows Promise for Friedreich’s Ataxia in Primate Study at MDA Conference
Capsida Biotherapeutics’ experimental gene therapy, CAP-004, for Friedreich’s ataxia (FA), has demonstrated promising preclinical results in nonhuman primates. Research presented by Capsida at the 2025 MDA Clinical & Scientific Conference showed that a single intravenous administration of CAP-004 successfully delivered its genetic payload to critical disease-relevant tissues, including the heart, sensory nerves, and the central nervous system (CNS), which encompasses the brain and spinal cord.
Notably, the therapy boosted levels of frataxin, the protein deficient in FA, in both the brain and heart to levels anticipated to be therapeutically significant for patients. Company data suggests CAP-004, utilizing a next-generation AAV capsid engineered for enhanced blood-brain barrier crossing, has the potential for near-complete correction across all CNS target tissues and is positioned as a “best-in-class” therapy to address the cardiac, CNS, and sensory manifestations of FA. The therapy delivers a functional version of the FXN gene, aiming to enable continuous frataxin production, thereby easing FA symptoms and slowing disease progression.
Non-human primate studies revealed that CAP-004 led to higher RNA expression in the brain, spinal cord, sensory nerve cells, and retina compared to AAV9, while also showing lower RNA expression in the liver, suggesting a reduced risk of liver toxicity. Furthermore, CAP-004 transduced approximately 80% of cells in CNS tissues and about 30% of the heart’s left ventricle. Importantly, the majority of the increased frataxin protein in the brain and heart was the mature, functional form, with brain levels exceeding untreated controls by over eight times and heart levels by about 1.7 times. These levels are projected to be efficacious in FA patients, and CAP-004 has been well-tolerated in preclinical studies. Ongoing research will focus on long-term durability and safety as the therapy advances towards clinical trials.
Source: https://www.mdaconference.org/abstract-library/systemic-aav-gene-therapy-with-next-generation-engineered-capsids-for-treatment-of-cns-and-cardiac-symptoms-in-friedreichs-ataxia/
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