PD-GBA is driven by mutations in the gene GBA1. This gene contains the instructions for making the lysosomal enzyme beta-glucocerebrosidase, or GCase, which is needed for the disposal and recycling of glycolipids — a type of cellular component that is known to accumulate with aging. PD-GBA patients have mutations in at least one chromosomal copy of GBA1, while Gaucher disease patients have mutations in both chromosomal copies. Without enough GCase, glycolipids accumulate, causing lysosomal dysfunction and aggregation of the protein α-Synuclein in cells, which is believed to lead to the inflammation and neurodegeneration behind PD-GBA.
PRV-PD101 delivers PR001, the well-studied viral vector AAV9 carrying a healthy copy of the GBA1 gene to the brain. This is administered by a one-time injection into an area above the spinal canal called the cisterna magna — a direct, non-surgical technique that has been used safely in humans for a century.
The U.S. FDA has granted Fast Track Designation for PR001 for the treatment of PD-GBA. It has also granted Orphan Drug Designation for PR001 for the treatment of patients with Gaucher disease.
PROPEL, the Phase 1/2 clinical trial of PR001 for the treatment of PD-GBA patients, is now recruiting as a multicenter, open-label, ascending dose, first in-human study. The purpose is to evaluate the safety of intracisternal PR001 administration in patients with moderate to severe Parkinson's disease with at least 1 pathogenic GBA1 mutation. Two escalating dose cohorts are planned (low dose and high dose).
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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