FDA Greenlights Capsida's CAP-003 Gene Therapy for Parkinson's

The U.S. Food and Drug Administration (FDA) has given the green light to Capsida Biotherapeutics for their investigational new drug (IND) application for CAP-003, a promising treatment for Parkinson’s disease. This approval allows the company to initiate clinical trials for CAP-003, specifically targeting Parkinson’s disease associated with glucocerebrosidase (GBA) mutations, known as PD-GBA. The approval marks a significant milestone for Capsida, being their second fully-owned clinical program to receive IND clearance.

CAP-003 is a gene therapy administered intravenously, designed to deliver genetic material across the blood-brain barrier with precision. The innovative therapy aims to minimize unintended effects on surrounding tissues. Capsida is preparing to launch a Phase I/II clinical trial for CAP-003, with plans to administer the first dose to a participant by the third quarter of 2025.

Swati Tole, the chief medical officer of Capsida Biotherapeutics, emphasized the critical need for new treatments in the realm of PD-GBA. Current treatment options fail to address the underlying issues caused by mutations in the GBA gene, which encodes the glucocerebrosidase enzyme, or GCase. As such, these mutations significantly heighten the risk of developing Parkinson’s disease, affecting up to 15% of patients. Research indicates that individuals with these mutations experience a 30% reduction in GCase enzyme activity compared to healthy individuals, yet no existing treatment effectively addresses this deficiency or halts disease progression.

Capsida’s approach seeks to fill this treatment void by offering a solution that targets the root of the problem, potentially slowing or even stopping the progression of the disease. As part of its broader mission, Capsida Biotherapeutics is building a pipeline focused on the central nervous system, with treatments intended for both rare and common neurological conditions affecting patients of all ages.

In summary, the FDA's approval of Capsida's IND application for CAP-003 represents a significant advancement in the search for effective treatments for Parkinson's disease linked to GBA mutations. It underscores the urgent need for therapies that go beyond symptom management to address the fundamental causes of the disease. As Capsida moves forward with clinical trials, there is hope that CAP-003 could provide a new avenue for patients who currently face limited treatment options.