Neurocrine, Voyager tout gene therapy clinical results for Parkinson's disease

By The Science Advisory Board staff writers

September 11, 2020 -- Neurocrine Biosciences and Voyager Therapeutics have released data from a phase Ib open-label, three-year efficacy and safety study of NBIb-1817, an investigational gene therapy to improve motor function in patients with Parkinson's disease.

NBIb-1817 is an investigational recombinant adeno-associated viral serotype 2 vector encoding the gene for human aromatic L-amino acid decarboxylase (AADC) that is designed to help produce the AADC enzyme in brain cells. The therapy helps restore the brain's ability to convert levodopa to dopamine and overcome deficits associated with Parkinson's disease.

The study found that one-time treatment with the gene therapy sustained improvement in motor function, including greater "on" time without dyskinesia (uncontrolled, involuntary muscle movement), a reduction in Unified Parkinson's Disease Rating Scale part III scores, and a reduction in the amount of medication patients required. The therapy was also well-tolerated with no serious adverse side effects.

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