New gene therapy for Parkinson’s disease – targeting GBA1 mutations

Parkinson’s disease is one of the most frequent disorders of brain. Affected individuals experience impairment of muscle control, which at later stages is often accompanied by cognitive problems. There is no cure for the disease. Known treatments control symptoms by modifying brain’s neurotransmitters (such as dopamine) and conductivity (through deep brain stimulation).

Scientists in a new clinical trial (NCT04127578) will target origin of Parkinson’s disease variant – GBA1 gene. Mutations in GBA1 are present in 3-7% patients. The study will recruit 16 individuals with at least one pathogenic mutation of GBA1.

Entire gene therapy will consist of single injection only. Physicians aim to administer AAV9 (adeno-associated viruses serotype 9) containing correct GBA1. The virus is hoped to establish long-term expression of GBA1 in neurons, leading to higher concentration of beta-glucocerebrosidase. Among possible effects, sponsor of the trial – Prevail Therapeutics – lists slowed neurodegeneration, reduced inflammation, and reduced aggregation of alfa-Synucleins.

The study will be completed in August 2026.

The same viral vector is planned to be used in another disorder caused by GBA1 mutations – rare Gaucher disease.

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