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PR001 gene therapy improved phenotypes in models of Parkinson’s disease with GBA1 mutation

Mutations in GBA1, which result in deficiency of lysosomal enzyme glucocerebrosidase (GCase), are the most common known genetic cause of Parkinson’s disease (PD). Decreased GCase activity in PD patients with GBA1 mutations (PD‐GBA) causes accumulation of glycolipid substrates, which leads to lysosomal dysfunction and neuroinflammation. With our gene therapy product candidate, PR001, we aim to increase GCase activity in PD‐GBA patients in order to ameliorate the lysosomal dysfunction and slow or stop disease progression.