VY-AADC01 gene therapy for Parkinson’s disease with various results

36 months after genetic treatment, 15 patients have maintained primary outcome of the therapy and possibly slightly improved their daily life.

VY-AADC01 gene therapy aimed to insert AADC gene directly into patient’s forebrain. Activity of the gene was expected to improve biochemical processing of dopamine molecules.

Clinical measurement of AADC activity and dopamine biochemistry proved that the therapy was effective at its primary goal. Patients have lowered doses of levodopa drugs.

Improvement of dopamine processing might help in Parkinson’s disease symptoms easing. However, only some patients reported subjective life improvement. Assessment of Unified Parkinson’s Disease Rating Scale pointed to slight recovery of movement abilities.

More: 143rd Annual Meeting of the American Neurological Association.