Capsida Biotherapeutics Receives FDA IND Clearance for CAP-003, a Novel AAV Gene Therapy for Parkinson’s Disease with GBA Mutations

THOUSAND OAKS, Calif. – June 11, 2025 – Capsida Biotherapeutics (“Capsida”) today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CAP-003. This potential best-in-class, intravenously (IV) administered adeno-associated virus (AAV) gene therapy is set to enter clinical trials for Parkinson’s disease associated with GBA mutations (PD-GBA). This marks Capsida’s second wholly-owned clinical program with a cleared IND, underscoring the company’s progress in AAV gene therapy development.

Both of Capsida’s clinical programs leverage a proprietary IV-delivered, blood-brain barrier-crossing engineered AAV capsid. This innovative delivery system is paired with proprietary cargo that is designed to avoid off-target tissues such as the liver and dorsal root ganglia (DRG), enhancing the therapy’s specificity and safety profile.

“PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression,” said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. “We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year.”

About CAP-003 and the Phase 1/2 Clinical Trial:

In non-human primate (NHP) studies, a single IV infusion of CAP-003 demonstrated dose-dependent increases in Glucocerebrosidase (GCase) activity in crucial brain regions, including the substantia nigra, frontal cortex, caudate nucleus, and putamen. These increases were substantially above the established 30% efficacy threshold expected to restore enzyme activity levels to normal in patients with PD-GBA. A Good Laboratory Practices (GLP) toxicology study in NHPs also showed a well-tolerated safety profile with no adverse histopathology. Capsida anticipates dosing the first patient in the Phase 1/2 clinical trial in the third quarter of this year. More information about the trial can be found at www.clinicaltrials.gov (NCT07011771).

About Parkinson’s Disease Associated with GBA Mutations (PD-GBA):

Mutations in GBA, the gene responsible for expressing the GCase enzyme, affect up to 15% of Parkinson’s patients, making it the most common genetic risk factor for the disease. Post-mortem studies have revealed an approximate 30% GCase activity deficit in patients compared to healthy individuals. Currently, there are no approved treatments that specifically target GCase, nor are there any approved disease-modifying treatments for Parkinson’s disease. Previous investigational treatments for PD-GBA have faced challenges due to their inability to adequately cross the blood-brain barrier and supplement GCase enzyme activity to overcome the deficit in patients and impact disease progression. This has often necessitated invasive direct brain or cerebrospinal fluid (CSF) administration, yielding limited results and imposing a significant burden on patients. CAP-003’s IV administration and blood-brain barrier-crossing capability, facilitated by its engineered AAV, offer a potentially transformative alternative.