The US Food and Drug Administration (FDA) has approved Capsida Biotherapeutics’ investigational new drug (IND) application for CAP-003 to treat Parkinson’s disease (PD).
The clearance allows CAP-003, an intravenously administered gene therapy, to proceed to clinical trials for treating PD associated with glucocerebrosidase (GBA) mutations (PD-GBA).
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This is Capsida’s second completely owned clinical programme that has successfully navigated through IND clearance.
CAP-003 leverages an engineered capsid capable of crossing the blood-brain barrier and delivering targeted genetic material while minimising off-target tissue effects.
The company plans to commence the Phase I/II study for CAP-003, with the first subject anticipated to receive the dose in the third quarter of 2025.
Capsida Biotherapeutics chief medical officer Swati Tole stated: “PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase [glucocerebrosidase], which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression.
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By GlobalData“We recognise 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.”
Mutations in GBA are prevalent in up to 15% of individuals with PD and pose the most significant genetic risk factor for developing the disease.
Studies have shown that patients exhibit around a 30% deficit in GCase enzyme activity compared to healthy subjects, yet no approved treatments address this issue or modify disease progression. Capsida has a central nervous system pipeline comprising disease-modifying and potentially curative treatments intended for rare as well as more common diseases across all ages.
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