Shanghai, China, May 14^th, 2025 —— Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that it has received National Medical Products Administration (NMPA) implied approval of an Investigational New Drug (IND) application for BBM-P002, the company’s Parkinson's Disease (PD) gene therapy candidate.

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, “We are pleased to share the good news of the NMPA’s implied clinical trial approval of BBM-P002. Building on its extensive experience in the research and production of gene therapies for rare diseases, BBM is actively expanding its development of gene therapies for chronic diseases to bring new treatment options to the patients. Utilizing an AAV vector to deliver genes essential for dopamine synthesis to the patient's putamen for sustained expression, BBM-P002 Injection holds the potential to achieve 'single-dose, long-term efficacy' effect for PD. We will accelerate the progress of clinical research to bring new treatment options to PD patients as early as possible.”

This clinical trial is an open label and single-dose clinical study, which was designed to evaluate the safety and efficacy of BBM-P002 Injection via stereotactic bilateral putamen injection in subjects with PD. Due to the engineered capsid independently developed by the BBM, BBM-P002 has a higher efficiency to transduce nervous tissue. Therefore, the proposed clinical dosage is lower than that of the similar adeno-associated virus (AAV) gene therapy candidates aboard.

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 patients¹. 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 phase². Current treatment methods for PD include pharmacotherapy, surgical interventions, botulinum toxin therapy, exercise therapy, psychological support and specialized care ³. 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 with inadequate symptom improvement from pharmacotherapy. 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).

Since February 2023, the Investigator Initiated Trial (IIT, NCT05822739) of BBM-P002 has been on-going. May 2025, BBM-P002 received NMPA’s implied approval of an IND application.

References

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

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

[3] 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 by the NMPA of 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.