ShanghaiJune 12, 2026 /PRNewswire/ — Belief BioMed (BBM), an innovative enterprise focused on cutting-edge gene therapy, today announced that the early-stage clinical study results of its independently developed and manufactured BBM-P002 for treating patients with moderate-to-advanced Parkinson’s disease have been officially published in the top-tier international academic journal Nature Medicine (IF=50), titled “Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial”1. This marks a milestone in China’s neurodegenerative disease gene therapy field, indicating that the original research has gained recognition from the international academic community. The study was conducted by the team led by Director Liu Jun at Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, and the team led by Director Shen Lu at Xiangya Hospital, Central South University, with clinicaltrials.gov registration number NCT05822739.
Background:
The BBM-P002 study is a multicenter, single-arm, open-label clinical trial evaluating safety and preliminary efficacy, enrolling 10 subjects across multiple dose groups. This study aims to assess the safety, tolerability, and efficacy of stereotactic bilateral putamen injection of BBM-P002 in treating moderate-to-advanced Parkinson’s disease. The follow-up is divided into a main study phase within 52 weeks post-administration and a long-term follow-up phase from 52 weeks to 5 years. All subjects have successfully completed the 52-week main study phase follow-up and entered the long-term follow-up phase. The published data represent interim results after all subjects completed the 52-week main study visit.
Results:
1. Favorable Safety Profile with Excellent Overall Tolerability
This study enrolled 10 subjects to systematically evaluate the safety and tolerability of BBM-P002 within 52 weeks post-administration.
- Follow-up data showed that all adverse events (AEs) occurring after BBM-P002 administration were mild, with no AEs related to the study drug; AEs related to the administration method included transient headache, weakness, etc., all resolving within a short period.
- During the 52-week study follow-up period, no dose-limiting toxicity (DLT) or serious adverse events (SAEs) occurred, and no systemic drug toxicity reactions were observed throughout.
2. Significant Efficacy in High-Dose Group with Notable Improvements in Multiple Core Indicators
The study focused on systematically evaluating the clinical efficacy at 52 weeks post-administration in 5 subjects in the high-dose group (1.2×10¹² vg).
- After high-dose BBM-P002 administration, all 5 subjects showed improvements in motor function. At the 12-month follow-up, the “off-state” MDS-UPDRS Part III score improved by an average of 21.6 points (46%) from baseline, and the “on-state” score improved by an average of 17.6 points (64%); the total MDS-UPDRS score also improved by 29.4 points (41%).
- In the high-dose group, patient diary data further confirmed clinical benefits, with daily “on-time” without dyskinesia increasing by 3.3 hours (40%) and total “on-time” increasing by 2.8 hours (29%).
- Alongside motor function improvements, ¹⁸F-FDOPA PET imaging provided exploratory evidence. Subjects showed restored dopaminergic neural pathway function in multiple key brain regions, with significantly enhanced tracer uptake signals in the bilateral dorsolateral putamen (dlPu) and bilateral globus pallidus (GP). Additionally, data analysis confirmed a significant correlation between the degree of PET signal enhancement in the bilateral dorsolateral putamen (dlPu) and improvements in patient motor function, providing imaging-level evidence supporting the therapeutic effect of BBM-P002.
Motor function and ¹⁸F-FDOPA PET imaging results for the high-dose group (1.2×10¹² vg)
Conclusion: The clinical study results indicate that all tested doses of BBM-P002 demonstrated good safety and tolerability, with preliminary clinical benefits observed in the high-dose group. These interim findings validate the clinical application value of BBM-P002, showing promising development prospects and application potential.
Supported by the scientific foundation of these early data, the BBM-P002 clinical trial (CXSL2500162 / NCT07195825), led by Director Liu Jun’s team at Ruijin Hospital, is progressing steadily. Director Liu Jun of Ruijin Hospital stated: “This work published in Nature Medicine reflects the ability of Chinese research teams to integrate safety monitoring, multidisciplinary collaboration, and clinical research standards in the cutting-edge field of neurological gene therapy. Subsequent registered clinical studies will further strengthen the evidence base.” Professor Xiao Xiao, co-founder, chairman, and chief scientist of Belief BioMed, said: “We sincerely thank the clinical experts and subjects for their outstanding contributions. This publication in Nature Medicine is a key milestone, powerfully validating the application potential of gene therapy in chronic diseases. We will continue to adhere to Good Clinical Practice (GCP) standards and work closely with clinical experts to steadily advance the development of this innovative therapy.”
About Parkinson’s Disease
Parkinson’s disease (PD) is the second most common neurodegenerative disease globally. In China, the prevalence of PD among people aged 60 and above is 1.37%, with approximately 3.62 million patients2. Its natural course can last up to 20 years, with core clinical symptoms including motor symptoms such as bradykinesia, resting tremor, muscle rigidity, and postural instability, as well as non-motor symptoms that may appear in the prodromal phase, such as constipation, hyposmia, REM sleep behavior disorder, depression, apathy, and cognitive and psychiatric disorders3. PD treatments include medication, surgery, botulinum toxin therapy, exercise therapy, psychological intervention, and nursing care4. Medication is the first-line and primary treatment, with levodopa as the preferred drug; patients may also opt for deep brain stimulation. However, both drugs and surgery offer limited symptom improvement and cannot slow disease progression or provide a cure.
With a deeper understanding of PD’s pathological mechanisms, gene therapy drugs for PD are gaining attention, with multiple candidates entering clinical trials. Based on disclosed data, gene therapy can improve clinical symptoms in PD patients to varying degrees, offering newer and more diverse treatment options.
About BBM-P002
BBM-P002 is an AAV gene therapy drug with proprietary intellectual property owned by Belief BioMed. Using stereotactic brain injection technology and an engineered AAV vector with high affinity for the nervous system, it delivers gene fragments encoding key enzymes required for dopamine production—tyrosine hydroxylase (TH) and aromatic L-amino acid decarboxylase (AADC)—to the striatum (primarily the putamen). This enables long-term, high-level expression of these key enzymes in the brain’s striatal region, continuously producing dopamine to treat Parkinson’s disease. It aims to achieve a “one-time administration, long-term efficacy” therapeutic effect. The production of BBM-P002 utilizes the company’s independently developed large-scale serum-free suspension culture process, compliant with Good Manufacturing Practice (GMP) requirements.