Capsida Receives FDA IND Clearance for Its IV-Administered Gene Therapy for Parkinson's Disease Associated With GBA Mutations
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for CAP-003, its potential best-in-class intravenously (IV) administered gene therapy, to enter clinical trials for Parkinson's disease associated with GBA mutations (PD-GBA). This is the second wholly owned clinical program developed by Capsida with a cleared IND. Both programs utilize a proprietary IV-delivered, blood brain barrier-crossing engineered capsid and proprietary cargo that is detargeted from off-target tissues, like liver and dorsal root ganglia (DRG). In addition, Capsida uses a proprietary manufacturing process and CAP-003 is manufactured in Capsida's state-of-the-art wholly owned Good Manufacturing Practice (GMP) facility.
'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 to date, a single IV infusion of CAP-003 resulted in dose-dependent increases in GCase activity in critical brain regions including the substantia nigra, frontal cortex, caudate nucleus, and putamen substantially above the established 30% efficacy threshold expected to restore enzyme activity levels back to normal in patients with PD-GBA. The NHP Good Laboratory Practices (GLP) toxicology study demonstrated a well-tolerated safety profile with no adverse histopathology. Capsida expects to dose the first patient in the Phase 1/2 clinical trial in the third quarter of this year. For more information about the Phase 1/2 clinical trial, please visit www.clinicaltrials.gov (NCT07011771).
About Parkinson's disease associated with GBA mutations (PD-GBA)
Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of Parkinson's patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients compared to healthy individuals 1. There are no approved treatments that target GCase and there are no approved disease modifying treatments for PD. Other investigational treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, those treatments have required invasive direct brain or cerebrospinal fluid (CSF) administration, with limited results, and a significant burden for patients.
About Capsida Biotherapeutics
Capsida Biotherapeutics is a clinical-stage, fully integrated next-generation genetic medicines company. It has 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 wholly owned pipeline includes its first-in-class investigational treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), CAP-002; and potential best-in-class investigational treatment for Parkinson's disease associated with GBA mutations (PD-GBA) CAP-003. Both have received FDA Investigational New Drug (IND) clearance to initiate clinical trials. Capsida's pipeline also includes a potential best-in-class treatment for Friedreich's ataxia (FA). In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. 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 www.capsida.com.
1 Leyns, C. E, G. et al (2023). npj Parkinson's Disease, 74(9).
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