Capsida Biotherapeutics announced it has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) to begin clinical trials of its intravenous (IV)-administered gene therapy for Parkinson’s disease associated with GBA mutations.
The California-based biotechnology company said that the Phase 1/2 trial will evaluate CAP-003, a gene therapy that uses a next-generation adeno-associated virus (AAV) capsid and a proprietary GBA1 transgene designed to increase glucocerebrosidase (GCase) activity. According to the company, GBA mutations are the most common genetic risk factor for Parkinson’s, impacting an estimated 10-15% of people with the disease.
The company claims CAP-003 has shown promising preclinical results, demonstrating widespread biodistribution in the brain and significant GCase enzyme activity following a single IV dose. The therapy aims to target both motor and non-motor symptoms of Parkinson’s disease.
“Receiving IND clearance for our lead program is a significant milestone,” said Peter Anastasiou, CEO of Capsida. He added that the company’s gene therapy platform is built for broad delivery to the central nervous system with cell-type specificity.
Capsida also reported that it is actively enrolling patients and preparing clinical trial sites in the U.S., with initial safety and biomarker data expected in the near term.
