Category: Parkinson’s Disease: Clinical Trials
Objective: To evaluate the 12-month safety, tolerability, and preliminary efficacy of BBM-P002, a novel dual-optimized gene therapy, in participants with moderate-to-advanced Parkinson’s disease (PD).
Background: Restoring striatal dopamine synthesis via gene therapy is a promising strategy for PD. BBM-P002 is a novel AAV vector (AAVT42) co-delivering a constitutively active, N-terminally truncated tyrosine hydroxylase (TH) variant and aromatic L-amino acid decarboxylase (AADC). This dual-optimization strategy is designed to enable continuous, high-efficiency dopamine production.
Method: In this multicenter, open-label, dose-escalation phase I trial, 10 participants with moderate-to-advanced PD received bilateral intraputaminal infusion of BBM-P002. Participants were enrolled into the lower-dose cohorts (ranging from 4.0 × 10¹¹ to 1.0 × 10¹² vg; n=5) or a high-dose cohort (1.2×10¹² vg; n=5). Primary outcomes were safety and tolerability. Secondary outcomes included motor function, patient diaries, and striatal dopamine synthesis assessed by ¹⁸F-FDOPA PET over a 12-month period.
Results: BBM-P002 was safe and well-tolerated, with no drug-related serious adverse events. At 12 months, the high-dose cohort demonstrated significant clinical improvements. The mean MDS-UPDRS Part III (motor) score improved by 21.6 points (46%) in the Off-medication state and by 17.6 points (64%) in the On-medication state (both p<0.001). Daily “good” ON time without troublesome dyskinesia increased by 3.3 hours (p<0.001). These motor gains were significantly correlated with increased ¹⁸F-FDOPA uptake in the putamen (r = -0.64, p=0.01). Improvements were also observed in non-motor domains including sleep, cognition, anxiety, and depression.
Conclusion: Intraputaminal delivery of BBM-P002 demonstrated a favorable safety profile and provided preliminary evidence of both motor and non-motor efficacy in patients with PD. These clinical benefits support the biological restoration of dopamine synthesis and warrant continued development in larger, randomized controlled trials.
To cite this abstract in AMA style:
M. Niu, J. Lin, J. Liu. A Phase I Multicenter Trial of a Novel Gene Therapy BBM-P002 for Parkinsons Disease [abstract]. Mov Disord. 2026; 41 (suppl 1). https://www.mdsabstracts.org/abstract/a-phase-i-multicenter-trial-of-a-novel-gene-therapy-bbm-p002-for-parkinsons-disease/. Accessed October 1, 2026.« Back to 2026 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/a-phase-i-multicenter-trial-of-a-novel-gene-therapy-bbm-p002-for-parkinsons-disease/