Belief BioMed's Duchenne Therapy BBM-D101 IND Approved by NMPA

Belief BioMed (BBM), a pioneering biotechnology enterprise based in Shanghai, China, has recently announced a significant milestone in its journey towards developing advanced gene therapies. The company has received approval from the National Medical Products Administration (NMPA) to initiate an Investigational New Drug (IND) application review for its Duchenne Muscular Dystrophy (DMD) gene therapy candidate, BBM-D101. This development follows an earlier clearance by the FDA, marking a crucial step in international regulatory acceptance.

Dr. Xiao Xiao, who is both a Co-founder and the Chairman and Chief Science Officer of Belief BioMed, expressed his delight at this achievement, coinciding with the Lunar New Year celebrations. Dr. Xiao emphasized the importance of collaborating with the NMPA to advance the IND review process for BBM-D101, which represents a beacon of hope for patients grappling with this challenging genetic disorder.

The impending clinical trial for BBM-D101 is designed as an open-label, single-dose study aimed at assessing the safety and efficacy of this therapy in young DMD patients aged between four and nine years. BBM-D101 harnesses an engineered capsid, enhancing its ability to effectively target and transduce muscle tissue. Consequently, the proposed dosage for this therapy is lower than that of similar approved adeno-associated virus (AAV) gene therapies, which is anticipated to contribute positively to its safety profile.

Duchenne Muscular Dystrophy is a severe muscle disease linked to mutations in the dystrophin gene and is inherited in an X-linked recessive manner. It affects approximately one in every 5,000 male infants globally, with nearly 30% of cases resulting from new genetic mutations. In the United States, the number of DMD patients is estimated to be under 50,000. The disease typically manifests symptoms before the age of six, progressively leading to muscle weakness and loss of walking ability by ages ten to twelve. By age 30, patients often succumb to cardiac or respiratory failure. This dire scenario underscores the pressing need for innovative therapeutic options.

BBM-D101 stands out as an AAV-based gene therapy underpinned by BBM's proprietary intellectual property. It offers a "one dose, long-term effect" treatment strategy by delivering a single intravenous administration of an optimized gene throughout the body's muscle tissue. This is facilitated by the company's independently developed 500L serum-free suspension culture process, which adheres to the stringent Good Manufacturing Practice of Medical Products (GMP) standards.

Since July 2024, BBM-D101 has been undergoing an Investigator Initiated Trial (IIT) with the identifier NCT06641895. The therapy received Orphan Drug Designation (ODD) and Pediatric Rare Disease Designation (RPDD) from the FDA in November 2024, with an IND application clearance following in January 2025.

Belief BioMed Inc. is a globally recognized biotech company specializing in the research, development, manufacturing, and clinical deployment of gene therapies. The company's mission is to offer groundbreaking and effective gene therapies for severe genetic and chronic conditions through its safe and efficient viral vector technology. BBM boasts a robust portfolio of vector technologies, including the HEK293 cell suspension serum-free culture process and comprehensive chromatography purification methods. Additionally, it has established a commercial platform for the production of gene therapy drugs and developed a comprehensive R&D pipeline addressing unmet clinical needs in various therapeutic areas such as hemophilia, DMD, Parkinson's disease, and osteoarthritis. Several of its product candidates have either entered clinical trials or submitted IND applications, including a gene therapy for adult hemophilia B patients, which has already gained acceptance by the NMPA in China.