Shanghai, China, January 22^th, 2025 —— Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that it has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for BBM-D101, the company’s Duchenne Muscular Dystrophy (DMD) gene therapy candidate.

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said, “At the beginning of 2025, we are pleased to have the FDA’s clearance to proceed into the clinic with BBM-D101, an independently developed and manufactured drug for treatment of DMD, which is another important milestone for BBM. We appreciate the FDA’s review of the IND and look forward to continuing to work in collaboration with the agency to carry out clinical trial.”

This clinical trial is an open label and single-dose clinical study, which was designed to evaluate the safety and efficacy of a single intravenous infusion in DMD patients aged 4-9 years old. Due to engineered capsid, BBM-D101 has a higher efficiency to transduce muscle tissue. Therefore, the proposed clinical dosage is lower than the approved similar adeno-associated virus (AAV) gene therapy products, and BBM-D101 is expected to have good safety.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked recessive muscle disease caused by mutations in the dystrophin gene. There is about 1 DMD patient per 5,000 male infants worldwide [1], and nearly 30% of them have new gene mutations [2]. The estimated number of DMD patients in the United States is less than 50,000 [3]. The symptoms of DMD usually appear before the age of 6 years old or even in infancy. Most patients gradually lose the ability to walk at the age of 10-12 years old and lose their lives due to cardiac and / or respiratory failure at the age of 30 years old [4]. Innovative treatment options are urgently needed.

About BBM-D101

BBM-D101 is an AAV-based gene therapy with independent intellectual property rights owned by BBM. Based on engineered AAV vector, it is single intravenously administered to deliver the optimized gene into muscles of whole body, thereby achieving "one dose, long-term effect" treatment of DMD. The production of BBM-D101 uses the 500L serum-free suspension culture process independently developed by the company, which meets the requirements of the Good Manufacturing Practice of Medical Products (GMP).

Since July 2024, the Investigator Initiated Trial (IIT, NCT06641895) of BBM-D101 has been on-going. November 2024, BBM-D101 was granted Orphan Drug Qualification (ODD) and Pediatric Rare Disease Qualification (RPDD) by the FDA.

[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10330733/

[2] ttps://www.ncbi.nlm.nih.gov/books/NBK482346/

[3] https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy

[4] https://www.genome.gov/Genetic-Disorders/Duchenne-Muscular-Dystrophy

 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 NDA of a gene therapy for the treatment of adult patients with hemophilia B, has been accepted by the NMPA of China. For more information, visit www.beliefbiomed.com.

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-D101 described herein has not been approved for marketing.