Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial - PubMed
3 hours ago
- #Parkinson's disease
- #gene therapy
- #clinical trial
- Dual-target gene therapy BBM-P002 targets enzymes TH and AADC for autonomous dopamine synthesis in Parkinson's disease.
- Multicenter phase 1 trial involved 10 participants across four dose cohorts, showing favorable safety and tolerability over 12 months.
- No dose-limiting toxicities or drug-related serious adverse events occurred; adverse events were mild, transient, and unrelated to therapy.
- Intraputaminal delivery was safe and well-tolerated, supporting further clinical development of the gene therapy approach.