Gene therapy On/off fluctuations
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Single intraputaminal infusion of OXB-102 lentiviral gene therapy was associated with ~40% improvement in UPDRS-III off-state scores and reduced oral levodopa requirements in a small, uncontrolled Phase 1/2 trial.
Cohort 1 (low dose, n=2) showed a 25-point mean improvement (42%) in UPDRS Part III off score at 3 months; cohort 2 (intermediate dose, n=4) showed a 21-point mean improvement (~40%) at 6 months with an average 21% reduction in levodopa equivalent daily dose and no serious treatment-related adverse events in either group.