Belief BioMed Receives IND Clearance for DMD Gene Therapy BBM-D101

Shanghai, China, April 18th, 2025 — Belief BioMed (BBM), a forward-thinking biotechnology firm, has announced a significant milestone in its quest to advance gene therapy treatments. The company received clearance from the National Medical Products Administration (NMPA) for its Investigational New Drug (IND) application concerning BBM-D101, a groundbreaking gene therapy candidate aimed at treating Duchenne Muscular Dystrophy (DMD). Previously, the U.S. Food and Drug Administration (FDA) had also approved the IND for BBM-D101.

Belief BioMed’s Co-founder, Chairman, and Chief Science Officer, Dr. Xiao Xiao, expressed great satisfaction with the NMPA's decision. He acknowledged the agency's thorough review process and looked forward to continued collaboration as the company moves forward with clinical trials. With the IND clearance also granted by the FDA, BBM is poised to expedite clinical research, aiming to provide new treatment options for DMD patients promptly.

The clinical trial planned for BBM-D101 is both open-label and single-dose, designed to assess the safety and effectiveness of a single intravenous infusion in DMD patients aged between four and nine years. Thanks to its specially engineered capsid, BBM-D101 is anticipated to have superior efficiency in transferring genetic material into muscle tissues. Consequently, the proposed clinical dose is lower than those of approved analogous adeno-associated virus (AAV) gene therapy products, enhancing its safety profile.

Duchenne Muscular Dystrophy is a severe muscle disease linked to mutations in the dystrophin gene, following an X-linked recessive pattern. The condition affects approximately one in every 5,000 male infants globally, with nearly 30% of cases attributed to new genetic mutations. In the United States, the number of DMD patients is estimated to be below 50,000. Symptoms typically emerge before the age of six, and most patients lose ambulatory ability between 10 and 12 years. Sadly, life expectancy is significantly reduced, with cardiac and respiratory failure often leading to death by around age 30. This underscores the urgent need for innovative treatment solutions.

BBM-D101 represents an innovative AAV-based gene therapy, uniquely owned by BBM. The therapy utilizes an engineered AAV vector to deliver an optimized gene to muscle tissues throughout the body via a single intravenous administration. This approach aims to achieve a "one dose, long-term effect" for treating DMD. BBM’s proprietary 500-liter serum-free suspension culture method is employed in the production of BBM-D101, adhering to the stringent Good Manufacturing Practice (GMP) standards for medical products.

Since July 2024, BBM-D101 has been undergoing an Investigator-Initiated Trial (IIT) under the identifier NCT06641895. In November 2024, the FDA recognized the potential of BBM-D101 by granting it Orphan Drug Designation and Pediatric Rare Disease Designation. Following this, in January 2025, the FDA cleared the IND application, and now, with the recent NMPA clearance, BBM is set to advance its clinical endeavors.

Belief BioMed Inc. is a global leader in the biotechnology sector, specializing in the research, development, and manufacturing of gene therapy products. The company is dedicated to offering innovative gene therapies for severe genetic and chronic conditions through safe and efficient viral vector technology. BBM has developed an array of vector technologies and established a commercial production platform for gene therapy drugs. Its extensive R&D pipeline addresses unmet clinical needs across various therapeutic areas, including hemophilia, DMD, Parkinson's disease, and osteoarthritis. BBM has successfully advanced several product pipelines through clinical trials and regulatory filings. Notably, its gene therapy for hemophilia B in adult patients has gained approval from China's NMPA.