THOUSAND OAKS, Calif., April 3, 2025 – Capsida Biotherapeutics (“Capsida”) announced new data from a three-month GLP toxicology study in non-human primates (NHPs) with CAP-003, its intravenously (IV) delivered gene therapy for Parkinson’s disease associated with GBA mutations (PD-GBA). CAP-003, enabled by Capsida’s proprietary engineered capsid and cargo, shows potential as a best-in-class treatment for PD-GBA. These data will be presented at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) (April 1-5, 2025, Vienna) and the American Academy of Neurology (AAN) annual meeting (April 5-9, 2025, San Diego). Capsida is on track to file an Investigational New Drug (IND) application in the second quarter of 2025.
GBA mutations, affecting up to 15% of PD patients and representing the most common genetic risk factor, result in an approximate 30% GCase enzyme deficit compared to healthy individuals1. Currently, there are no approved treatments targeting GCase or disease-modifying treatments for PD. Other investigational PD-GBA treatments have faced limitations due to lack of specificity, inability to cross the blood-brain barrier, and insufficient GCase enzyme supplementation, often requiring invasive direct brain or cerebrospinal fluid (CSF) administration with limited success and significant patient burden.
In the study, CAP-003 was delivered via IV infusion at doses of 9.8E12, 3.1E13, or 5E13 vg/kg. Three months post-administration, results demonstrated dose-dependent increases in GCase activity in key brain regions, including the substantia nigra, frontal cortex, caudate nucleus, and putamen, exceeding the 30% efficacy threshold expected to restore normal enzyme activity levels in PD-GBA patients. A single IV infusion of CAP-003 increased average brain GCase activity by up to 206% and average brain GCase bulk protein by up to 415% in relevant brain regions compared to untreated animals. Strong correlation between CSF GCase protein and brain GCase activity supports the use of CSF GCase levels as a potential clinical biomarker. Furthermore, decreased plasma glucosylsphingosine levels confirm target engagement by CAP-003 in healthy NHPs and its utility as a clinical biomarker. CAP-003 was well tolerated with no adverse clinical or histopathological findings.
“We’re excited by the emerging data from our GLP toxicology study demonstrating the potential of CAP-003 as a best-in-class treatment for PD-GBA,” said Peter Anastasiou, Capsida’s Chief Executive Officer. “Capsida is on track to file an IND for CAP-003 in the second quarter of this year, with the goal of bringing a much-needed disease-modifying treatment option to those suffering from this debilitating disease.”
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