Shanghai, China, June 10, 2026 — Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the data from the early stage study for BBM-P002 with  advanced Parkinson's Disease, a gene therapy drug independently developed and manufactured by BBM, have been published in the authoritative international journal Nature Medicine(Impact Factor: 50.0). The paper is titled "Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial" ¹. This study was conducted under the leadership of Professor Jun Liu from Ruijin Hospital, Shanghai Jiao Tong University School of Medicine and Professor Lu Shen from Xiangya Hospital of Central South University. Clinicalrials.gov registration NCT05822739.

Background

The early stage  study of BBM-P002 is a multicenter, single-arm, open-label clinical trial involving 10 participants, with different cohort （low，middle，high）. To evaluate the safety, tolerability, and efficacy of stereotactic bilateral putamen injection of BBM-P002 for treating mid-to-late stage Parkinson's disease. The Follow-up periods include a main study within 52 weeks post-treatment and a long-term follow-up after week 52 till 5 years. All participants have now completed the 52-week main study follow-up and have entered the long-term follow-up. The data published in Nature Medicine at this time was the outcomes from all participants who completed the 52-week main study period.

Results

1. The safety results are good and the overall tolerance is excellent.

The study evaluate the safety of all 10 participants within 52 weeks after BBM-P002 treatment.

Ÿ   The data shows that all adverse events (AEs) that occurred after BBM-P002 treatment were mild，there were no AEs related to the study drug. The AEs related to the route of administration visit such as headaches and weakness etc, which all recovered within a short period of time.

Ÿ   No dose-limiting toxicity (DLT), no serious adverse events (SAE), and no systemic toxicity were observed within 52 weeks follow up.

2. Efficacy: The high-dose cohort showed remarkable therapeutic effects, with significant improvements achieved in multiple core indicators.

The efficacy of high-dose cohort (1.2×10¹² vg) was mainly evaluated in 5 participants within 52 weeks after BBM-P002 treatment.

Ÿ   All the participants (5/5) met the responder criterion at 12 months. At the 12-month follow-up, Off-state MDS-UPDRS Part III scores showed a mean improvement of 21.6 points from baseline (-46%), and On-state scores improved by a mean of 17.6 points (64%). The total MDS-UPDRS score also improved significantly by 29.4 points (41%).

Ÿ   These motor gains were further corroborated by patient-reported diaries. Daily ON time without dyskinesia increased by 3.3 hours (40%), and total ON time increased by 2.8 hours (29%).

Ÿ   Parallel to motor function improvements, ¹⁸F-FDOPA PET imaging provided exploratory evidence of dopaminergic restoration across regions, characterized by significantly increased tracer uptake signal in the bilateral dorsolateral putamen (dlPu) and bilateral globus pallidus (GP), from baseline to month 12. Notably, dlPu PET signal was significantly correlated with motor assessments. The therapeutic effect of BBM-P002 was confirmed from the imaging level.

Figure: Assessment of motor functions and ¹⁸F-FDOPA PET imaging outcomes in high dose cohort ( 1.2×10¹²vg )

Conclusions

All dose cohort in the IIT study had good safety and tolerability, and preliminary clinical benefits were observed in the high-dose cohort. These previous study support the clinical potential of the continued development of BBM-P002.

About Parkinson's Disease

Parkinson's disease (PD) is the second most common neurodegenerative diseases globally. In China, the prevalence of PD among individuals aged 60 and above is 1.37%, with approximately 3.62 million patients ². The natural course of PD can span up to 20 years, characterized by core clinical symptoms such as motor manifestations (bradykinesia, resting tremor, muscle rigidity, postural instability, etc.) and non-motor symptoms (constipation, hyposmia, REM sleep behavior disorder, depression, apathy, cognitive and psychiatric disturbances, etc.) that may emerge during the prodromal phase ³. Current treatment methods for PD include pharmacotherapy, surgical interventions, botulinum toxin therapy, exercise therapy, psychological support and specialized care ⁴. The first line and primary treatment method is pharmacotherapy, and the levodopa is the preferred drug. Deep brain stimulation is an additional treatment option for patients. However, both drugs and surgery offer limited symptomatic relief, failing to prevent disease progression or provide a cure.

With increasing understanding of the pathological mechanisms of PD, gene therapy for PD is gradually gaining more attention, and there are several gene therapy candidates that have entered the clinical research stage. Data disclosed to date indicates that the clinical symptoms of PD patients were alleviated to varying degrees by gene therapies, which offer novel treatment options for PD.

About BBM-P002

BBM-P002 is an AAV-based gene therapy with independent intellectual property rights owned by BBM. Using stereotactic brain injection technology and engineered AAV vectors with high nervous tissue tropism, the optimized gene expression cassettes are delivered to the bilateral putamen of the brain, enabling long-term and high-level expression of genes required for dopamine synthesis in the striatal region. This therapy offers the potential to achieve a "single-dose, long-lasting efficacy" efficacy. The production of BBM-P002 uses the large-scale serum-free suspension culture process independently developed by the BBM, which meets the requirements of the Good Manufacturing Practice of Medical Products (GMP).

This early stage study data publicized in Nature Medicine provides new scientific support for gene therapy in PD. BBM will continue to advance the clinical program, aiming to accelerate the availability of this innovative treatment option to PD patients.

References

[1] Mengyue Niu, et al. Nat Med. 2026 Jun 10. https://doi.org/10.1038/s41591-026-04436-0.

[2] Kalia LV, et al. Lancet. 2015 Aug 29;386:896-912. 

[3] Armstrong MJ, et al. JAMA. 2020 Feb 11;323(6):548-560.

[4] Clinical Guidelines for Parkinson's Disease Management (4th Edition). Chinese Journal of Neurology. 2020.53(12):973-786.

About Belief BioMed                                                                                            

Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson's disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The Biologics License Application (BLA) of a gene therapy for the treatment of adult patients with hemophilia B has been approved in Mainland China and Macao China.

Statement

This information is only for the purpose of introducing the company's event and information on that date, and is not intended to promote any company's products and/or services, nor should it be construed as providing any advice or recommendation on the selection of any drugs, medical devices and treatment options.

For information about any company products, diseases and/or treatments, please consult a healthcare professional.

BBM-P002 described herein has not been approved for marketing.