Capsida Biotherapeutics reported new preclinical data in non-human primates (NHP) that demonstrate the potential of its next-generation intravenously (IV) administered gene therapy candidate, CAP-003, to safely and effectively treat Parkinson’s disease (PD) associated with GBA mutations (PD-GBA). Collectively, the findings strongly support the best-in-class profile of CAP-003 and Capsida’s plans to advance the therapy into clinical trials in the first half of 2025, according to the company.

Mutations in GBA, the gene expressing the GCase enzyme, are the most common genetic risk factor for PD. Other 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 impact the disease.

In an attempt to overcome these challenges, they have utilized invasive direct brain or cerebrospinal fluid administration, with limited results, but significant burden for patients, explained a Capsida spokesperson, who added that Capsida’s wholly owned novel gene therapy offers the potential to supplement the GCase enzyme with a single IV infusion, enabling long-term disease modification and substantially slowing disease progression.

In non-human primates (NHPs), CAP-003 transduced the majority of neurons, including sub-cortical regions, resulting in GCase enzyme activity levels that exceed the threshold anticipated to restore GCase function in patients with PD-GBA. CAP-003 also demonstrated significant liver detargeting compared with AAV9 with no clinical pathology, immunogenicity, or histopathology findings, including in dorsal root ganglia (DRGs).

“Our novel wholly owned PD-GBA gene therapy demonstrates the potential to provide long-term significant decrease in disease progression safely with a single IV infusion,” said Capsida CEO Peter Anastasiou. “We look forward to advancing this best-in-class program into clinical development in the first half of 2025, so that we can bring this important treatment option to people suffering from this devastating disease.”

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