NHP data demonstrate potential for CAP-004 to treat CNS, cardiac, and sensory manifestations of Friedreich's ataxia

THOUSAND OAKS, Calif., Nov. 12, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") 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."

Details about the CAP-004 ICAR poster presentation:

Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich's Ataxia
Date: Wednesday, November 13, 2024, 6:00 PM GMT
Session: Emerging and existing therapeutics – preclinical research
Poster Number: 477
Location: Leonardo Royal Hotel London Tower Bridge, Minories Suite
Presenter: Celeste Stephany, Ph.D., Director of CNS and Ophthalmology Preclinical Research, Capsida

About Capsida Biotherapeutics
Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including a potential first-in-class treatment for STXBP1 developmental and epileptic encephalopathy and a best-in-class treatment Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. Capsida's third program is a potential best-in-class therapy for Friedreich's ataxia and is in IND-enabling studies. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at .

SOURCE Capsida Biotherapeutics