New Data Demonstrate Substantial Therapeutic Potential of Capsida's IV Gene Therapy for Parkinson's Disease

Capsida Biotherapeutics recently shared promising new data on its gene therapy, CAP-003, which is designed to treat Parkinson's disease linked to GBA mutations (PD-GBA). This gene therapy, delivered through an IV, shows strong potential for being safe and effective. It works by significantly increasing levels of the GCase protein, which is deficient in many Parkinson's patients with GBA mutations. Additionally, CAP-003 avoids affecting the liver and other sensitive areas, which is a common issue with other treatments. Mutations in the GBA gene, which affect about 15% of Parkinson's patients, are the most common genetic risk factor for the disease. These mutations lead to a deficiency in the GCase enzyme, which is important for brain health. Traditional treatments have struggled to provide enough GCase to make a difference, often requiring invasive methods to deliver the enzyme directly to the brain, with limited success and a heavy burden on patients. Capsida's research on animals showed that CAP-003, delivered via IV, greatly increased the amount of GCase enzyme in the brain—up to 250% higher in key regions compared to untreated animals. In contrast to other therapies, CAP-003 also reduced unwanted exposure to the liver and other areas by up to 19 times, while showing no safety concerns in the animal studies. According to Capsida's CEO, Peter Anastasiou, these results suggest that CAP-003 could safely restore GCase activity in Parkinson's patients with a single IV dose, potentially slowing the progression of the disease. The company expects to start human trials in 2025.