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Phase 1 Trial Shows Dual-Target Gene Therapy Safe for Parkinson's Disease
Key Takeaways
- Phase 1 trial of BBM-P002, a dual-target gene therapy co-delivering TH and AADC, shows safety and tolerability in 10 patients with moderate-to-advanced Parkinson's disease.
- No dose-limiting toxicities or drug-related serious adverse events were reported; 23 mild, transient adverse events were deemed unrelated to treatment.
- The therapy aims to enable autonomous dopamine synthesis, potentially reducing dependence on levodopa.
- Results support continued clinical development; efficacy data are pending.
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DT Editorial Team··via nature.com